David A Williams

Q: What Schools Are Affiliated With David A Williams

David A Williams is affiliated with the following schools: Harvard University, University of Cincinnati, University of California, Berkeley, Indiana University Bloomington, Indiana University, Hannover Medical School, University of California, San Francisco

David A Williams's AcademicInfluence.com Rankings

David A Williams

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#2627

World Rank

#3268

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Common Law

#120

World Rank

#129

Historical Rank

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David A Williams's Degrees

Bachelors Biology University of California, Berkeley
Doctorate Medicine Harvard University

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David A Williams's Published Works

Number of citations in a given year to any of this author's works

Total number of citations to an author for the works they published in a given year. This highlights publication of the most important work(s) by the author

Published Works

Haematopoietic stem cells do not transdifferentiate into cardiac myocytes in myocardial infarcts (2004) (2253)
Stem cell factor is encoded at the SI locus of the mouse and is the ligand for the c-kit tyrosine kinase receptor (1990) (1465)
Mouse model of X–linked chronic granulomatous disease, an inherited defect in phagocyte superoxide production (1995) (918)
Identification of primitive human hematopoietic cells capable of repopulating NOD/SCID mouse bone marrow: Implications for gene therapy (1996) (825)
Colocalization of retrovirus and target cells on specific fibronectin fragments increases genetic transduction of mammalian cells (1996) (562)
Hematopoietic Cell Regulation by Rac1 and Rac2 Guanosine Triphosphatases (2003) (533)
Fibronectin and VLA-4 in haematopoietic stem cell–microenvironment interactions (1991) (507)
Exit from dormancy provokes DNA-damage-induced attrition in haematopoietic stem cells (2015) (482)
Modulating the stem cell niche for tissue regeneration (2014) (481)
Rac1 Activation Controls Nuclear Localization of β-catenin during Canonical Wnt Signaling (2008) (477)
Introduction of new genetic material into pluripotent haematopoietic stem cells of the mouse (1984) (456)
Side effects of retroviral gene transfer into hematopoietic stem cells. (2003) (438)
Hematopoietic Stem‐Cell Gene Therapy for Cerebral Adrenoleukodystrophy (2017) (323)
A modified γ-retrovirus vector for X-linked severe combined immunodeficiency. (2014) (312)
Diffusion Dynamics of Glycine Receptors Revealed by Single – Quantum Dot Tracking (2012) (304)
Rac GTPases differentially integrate signals regulating hematopoietic stem cell localization (2005) (296)
Critical Roles for Rac1 and Rac2 GTPases in B Cell Development and Signaling (2003) (287)
Germline ETV6 mutations in familial thrombocytopenia and hematologic malignancy (2015) (284)
Efficient retrovirus-mediated transfer of the multidrug resistance 1 gene into autologous human long-term repopulating hematopoietic stem cells (2000) (282)
Highly efficient therapeutic gene editing of human hematopoietic stem cells (2019) (263)
Chance or necessity? Insertional mutagenesis in gene therapy and its consequences. (2003) (234)
The Public Repository of Xenografts Enables Discovery and Randomized Phase II-like Trials in Mice. (2016) (213)
mTORC1-dependent and -independent regulation of stem cell renewal, differentiation, and mobilization (2008) (192)
Genetic and Biochemical Evidence That Haploinsufficiency of the Nf1 Tumor Suppressor Gene Modulates Melanocyte and Mast Cell Fates in Vivo (2000) (188)
Equal potency of gammaretroviral and lentiviral SIN vectors for expression of O6-methylguanine-DNA methyltransferase in hematopoietic cells. (2006) (180)
Genetic Deletion of Rac1 GTPase Reveals Its Critical Role in Actin Stress Fiber Formation and Focal Adhesion Complex Assembly* (2006) (168)
Rac guanosine triphosphatases represent integrating molecular therapeutic targets for BCR-ABL-induced myeloproliferative disease. (2007) (157)
Differential niche and Wnt requirements during acute myeloid leukemia progression. (2011) (154)
Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency. (2008) (153)
Neurofibromin-deficient Schwann cells secrete a potent migratory stimulus for Nf1+/- mast cells. (2003) (150)
Localization of Rac2 via the C terminus and aspartic acid 150 specifies superoxide generation, actin polarity and chemotaxis in neutrophils (2004) (148)
Rac2-MRC-cIII-generated ROS cause genomic instability in chronic myeloid leukemia stem cells and primitive progenitors. (2012) (148)
Neurofibromin-deficient Schwann cells secrete a potent migratory stimulus for Nf1+/– mast cells (2003) (147)
Gene Therapy--New Challenges Ahead (2003) (145)
Impaired FANCD2 monoubiquitination and hypersensitivity to camptothecin uniquely characterize Fanconi anemia complementation group M. (2009) (142)
Overcoming reprogramming resistance of Fanconi anemia cells. (2012) (140)
Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease. (2020) (139)
Depletion of Jak2V617F myeloproliferative neoplasm-propagating stem cells by interferon-α in a murine model of polycythemia vera. (2013) (137)
Hyperactivation of P21ras and the Hematopoietic-Specific Rho Gtpase, Rac2, Cooperate to Alter the Proliferation of Neurofibromin-Deficient Mast Cells in Vivo and in Vitro (2001) (135)
The Rac2 Guanosine Triphosphatase Regulates B Lymphocyte Antigen Receptor Responses and Chemotaxis and Is Required for Establishment of B-1a and Marginal Zone B Lymphocytes1 (2002) (135)
Lentiviral gene therapy for X-linked chronic granulomatous disease (2020) (126)
TNF-α induces leukemic clonal evolution ex vivo in Fanconi anemia group C murine stem cells (2007) (124)
Serial transplantation of methotrexate-resistant bone marrow: protection of murine recipients from drug toxicity by progeny of transduced stem cells (1990) (124)
High-Efficiency Gene Transfer into Normal and Adenosine Deaminase-Deficient T Lymphocytes Is Mediated by Transduction on Recombinant Fibronectin Fragments (1998) (124)
Human RHOH deficiency causes T cell defects and susceptibility to EV-HPV infections. (2012) (123)
Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype. (2016) (117)
Comparison of three retroviral vector systems for transduction of nonobese diabetic/severe combined immunodeficiency mice repopulating human CD34+ cord blood cells. (2003) (116)
Signaling and cytoskeletal requirements in erythroblast enucleation. (2012) (115)
Inflammatory ROS promote and cooperate with the Fanconi anemia mutation for hematopoietic senescence (2007) (114)
Toxicity and Response following CD19-specific CAR T cells in pediatric/young adult relapsed/refractory B-ALL. (2019) (109)
Genomic analysis of bone marrow failure and myelodysplastic syndromes reveals phenotypic and diagnostic complexity (2015) (107)
Rac GTPases regulate the morphology and deformability of the erythrocyte cytoskeleton. (2006) (107)
RhoH GTPase recruits and activates Zap70 required for T cell receptor signaling and thymocyte development (2006) (106)
Biochemical and Biological Characterization of a Human Rac2 GTPase Mutant Associated with Phagocytic Immunodeficiency* (2001) (105)
Methylguanine methyltransferase-mediated in vivo selection and chemoprotection of allogeneic stem cells in a large-animal model. (2003) (105)
Rac GTPase isoforms Rac1 and Rac2 play a redundant and crucial role in T-cell development. (2008) (102)
Lentiviral vectors pseudotyped with murine ecotropic envelope: increased biosafety and convenience in preclinical research. (2006) (100)
The Apc(min) mouse has altered hematopoietic stem cell function and provides a model for MPD/MDS. (2010) (100)
Rac1 and a GTPase-activating protein, MgcRacGAP, are required for nuclear translocation of STAT transcription factors (2006) (100)
Stem Cell Collection and Gene Transfer in Fanconi Anemia. (2007) (100)
Genetic deletion of Cdc42GAP reveals a role of Cdc42 in erythropoiesis and hematopoietic stem/progenitor cell survival, adhesion, and engraftment. (2006) (99)
The role of chemokine activation of Rac GTPases in hematopoietic stem cell marrow homing, retention, and peripheral mobilization. (2006) (97)
Stem Cell Factor, Interleukin-3, and Interleukin-6 Promote Retroviral-Mediated Gene Transfer Into Murine Hematopoietic Stem Cells (1992) (97)
Structure-function based design of small molecule inhibitors targeting Rho family GTPases. (2006) (95)
miRNA-embedded shRNAs for Lineage-specific BCL11A Knockdown and Hemoglobin F Induction. (2015) (95)
Phenotypic correction of primary Fanconi anemia T cells with retroviral vectors as a diagnostic tool. (2002) (93)
TNF-alpha induces leukemic clonal evolution ex vivo in Fanconi anemia group C murine stem cells. (2007) (92)
Rac deletion in osteoclasts causes severe osteopetrosis (2011) (92)
Cdc42 critically regulates the balance between myelopoiesis and erythropoiesis. (2007) (91)
The FIP1L1-PDGFRA fusion gene cooperates with IL-5 to induce murine hypereosinophilic syndrome (HES)/chronic eosinophilic leukemia (CEL)-like disease. (2006) (90)
Inhibition of RhoA GTPase activity enhances hematopoietic stem and progenitor cell proliferation and engraftment. (2006) (90)
Rational design and applications of a Rac GTPase-specific small molecule inhibitor. (2006) (89)
Inflammatory Reactive Oxygen Species-Mediated Hemopoietic Suppression in Fancc-Deficient Mice1 (2007) (88)
Stem cell collection and gene transfer in Fanconi anemia. (2007) (88)
RAC2 loss-of-function mutation in 2 siblings with characteristics of common variable immunodeficiency. (2015) (84)
A rapid method for retrovirus-mediated identification of complementation groups in Fanconi anemia patients. (2005) (83)
Cross-talk between RhoH and Rac1 in regulation of actin cytoskeleton and chemotaxis of hematopoietic progenitor cells. (2008) (81)
Genetic evidence for convergence of c-Kit- and alpha4 integrin-mediated signals on class IA PI-3kinase and the Rac pathway in regulating integrin-directed migration in mast cells. (2003) (79)
Cross-talk between α4β1/α5β1 and c-Kit results in opposing effect on growth and survival of hematopoietic cells via the activation of focal adhesion kinase, mitogen-activated protein kinase, and Akt signaling pathways (2001) (78)
Rac GTPases in Human Diseases (2010) (73)
Sleeping beauty vector system moves toward human trials in the United States. (2008) (71)
Telomerase inhibition effectively targets mouse and human AML stem cells and delays relapse following chemotherapy. (2014) (71)
Rho GTPases and regulation of hematopoietic stem cell localization. (2008) (70)
Hematopoietic-specific Rho GTPases Rac2 and RhoH and human blood disorders. (2013) (67)
Rac2‐Deficient Hematopoietic Stem Cells Show Defective Interaction with the Hematopoietic Microenvironment and Long‐Term Engraftment Failure (2005) (66)
Rac2 Regulates Neutrophil Chemotaxis, Superoxide Production, and Myeloid Colony Formation through Multiple Distinct Effector Pathways1 (2005) (66)
Rac2‐deficient mice display perturbed T‐cell distribution and chemotaxis, but only minor abnormalities in TH1 responses (2002) (65)
Upping the ante: recent advances in direct reprogramming. (2009) (65)
Fanconi anemia deficiency stimulates HPV-associated hyperplastic growth in organotypic epithelial raft culture (2008) (64)
Rac2, a Hematopoiesis-Specific Rho GTPase, Specifically Regulates Mast Cell Protease Gene Expression in Bone Marrow-Derived Mast Cells (2002) (64)
Inhibition of Rac GTPase signaling and downstream prosurvival Bcl-2 proteins as combination targeted therapy in MLL-AF9 leukemia. (2011) (63)
Rac1 is essential for intraembryonic hematopoiesis and for the initial seeding of fetal liver with definitive hematopoietic progenitor cells. (2008) (63)
Stromal cell-associated hematopoiesis : immortalization and characterization of a primate bone marrow-derived stromal cell line (1991) (61)
Rac1 and Rac2 GTPases are necessary for early erythropoietic expansion in the bone marrow but not in the spleen (2010) (60)
Mechanisms underlying genetic susceptibility to multisystem inflammatory syndrome in children (MIS-C) (2021) (59)
Rac2 GTPase deficiency depletes BCR-ABL+ leukemic stem cells and progenitors in vivo. (2010) (59)
Evolving Gene Therapy in Primary Immunodeficiency. (2017) (59)
Rac1 GTPase Regulates Cell Genomic Stability and Senescence* (2006) (58)
Systematic analysis of the ability of stromal cell lines derived from different murine adult tissues to support maintenance of hematopoietic stem cells in vitro (1990) (58)
Role of p38 and ERK MAP kinase in proliferation of erythroid progenitors in response to stimulation by soluble and membrane isoforms of stem cell factor. (2002) (58)
Hematoprotection and enrichment of transduced cells in vivo after gene transfer of MGMTP140K into hematopoietic stem cells (2002) (56)
Interleukin-11 inhibits adipogenesis and stimulates myelopoiesis in human long-term marrow cultures (1993) (56)
Murine interleukin‐11 (IL‐11) is expressed at high levels in the hippocampus and expression is developmentally regulated in the testis (1996) (55)
Radiation-free, alternative-donor HCT for Fanconi anemia patients: results from a prospective multi-institutional study. (2017) (53)
The Public Repository of Xenografts Enables Discovery and Randomized Phase II-like Trials in Mice. (2016) (53)
Involvement of RhoH GTPase in the Development of B-Cell Chronic Lymphocytic Leukemia (2009) (52)
In vivo selection of human hematopoietic cells in a xenograft model using combined pharmacologic and genetic manipulations. (2003) (51)
RhoH is critical for cell-microenvironment interactions in chronic lymphocytic leukemia in mice and humans. (2012) (51)
The TRQQKRP motif located near the C-terminus of Rac2 is essential for Rac2 biologic functions and intracellular localization. (2002) (49)
Successful hematopoietic stem cell mobilization and apheresis collection using plerixafor alone in sickle cell patients. (2018) (48)
CXCL9 inhibits eosinophil responses by a CCR3- and Rac2-dependent mechanism. (2005) (46)
Stem cell gene therapy for fanconi anemia: report from the 1st international Fanconi anemia gene therapy working group meeting. (2011) (45)
The pseudokinase MLKL activates PAD4-dependent NET formation in necroptotic neutrophils (2018) (44)
RhoH GTPase: A Key Regulator of Hematopoietic Cell Proliferation and Apoptosis? (2005) (44)
Guanine nucleotide exchange factor Vav1 regulates perivascular homing and bone marrow retention of hematopoietic stem and progenitor cells (2011) (44)
Aberrant overexpression of CD14 on granulocytes sensitizes the innate immune response in mDia1 heterozygous del(5q) MDS. (2014) (43)
Vav3 collaborates with p190-BCR-ABL in lymphoid progenitor leukemogenesis, proliferation, and survival. (2012) (42)
Simultaneous Infection with Retroviruses Pseudotyped with Different Envelope Proteins Bypasses Viral Receptor Interference Associated with Colocalization of gp70 and Target Cells on Fibronectin CH-296 (1999) (40)
Defective homing is associated with altered Cdc42 activity in cells from patients with Fanconi anemia group A. (2008) (40)
Rho GTPases in hematopoietic stem cell functions (2009) (39)
Creation of a Fully Functional Human Chimeric DNA Repair Protein (1998) (39)
Medicine. Gene therapy--new challenges ahead. (2003) (39)
Leukaemia: Niche retreats for stem cells (2006) (38)
Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium study (2019) (37)
Hematopoietic expression of O(6)-methylguanine DNA methyltransferase-P140K allows intensive treatment of human glioma xenografts with combination O(6)-benzylguanine and 1,3-bis-(2-chloroethyl)-1-nitrosourea. (2003) (35)
The Rac GTPase effector p21-activated kinase is essential for hematopoietic stem/progenitor cell migration and engraftment. (2013) (35)
Stem cell transplants for patients with X-linked agammaglobulinemia. (2003) (35)
Child Health Research Funding and Policy: Imperatives and Investments for a Healthier World (2010) (34)
A theoretically optimized method for cord blood stem cell cryopreservation. (2003) (34)
Rapid Lentiviral Transduction Preserves the Engraftment Potential of Fanca-/- Hematopoietic Stem Cells. (2008) (33)
Loss of Function of TET2 Cooperates with Constitutively Active KIT in Murine and Human Models of Mastocytosis (2014) (33)
Identification and characterization of mutations in FANCL gene: A second case of Fanconi anemia belonging to FA‐L complementation group (2009) (32)
Suppressive effects in vivo of purified recombinant human H-subunit (acidic) ferritin on murine myelopoiesis (1989) (32)
RhoH Regulates Subcellular Localization of ZAP-70 and Lck in T Cell Receptor Signaling (2010) (31)
Live and let die: in vivo selection of gene-modified hematopoietic stem cells via MGMT-mediated chemoprotection. (2007) (31)
Reciprocal relationship between O6-methylguanine-DNA methyltransferase P140K expression level and chemoprotection of hematopoietic stem cells. (2008) (31)
Finding the needle in the hay stack: hematopoietic stem cells in Fanconi anemia. (2009) (30)
Signaling Through the Interaction of Membrane-Restricted Stem Cell Factor and c-kit Receptor Tyrosine Kinase: Genetic Evidence for a Differential Role in Erythropoiesis (1998) (30)
PTEN Negatively Regulates Engulfment of Apoptotic Cells by Modulating Activation of Rac GTPase (2011) (30)
RAC2 GTPase deficiency and myeloid cell dysfunction in human and mouse. (2002) (28)
Gene Therapy 2000. (2000) (28)
Promoting Translational Research in Academic Health Centers: Navigating the “Roadmap” (2005) (28)
A key role for Rac and Pak signaling in neutrophil extracellular traps (NETs) formation defines a new potential therapeutic target (2018) (28)
Ectopic HOXB4 overcomes the inhibitory effect of tumor necrosis factor-{alpha} on Fanconi anemia hematopoietic stem and progenitor cells. (2009) (28)
T-cell growth factor P40 promotes the proliferation of myeloid cell lines and enhances erythroid burst formation by normal murine bone marrow cells in vitro (1990) (27)
RAC reviews serious adverse event associated with AAV therapy trial. (2007) (27)
Importance of murine study design for testing toxicity of retroviral vectors in support of phase I trials. (2007) (27)
T cell dynamics and response of the microbiota after gene therapy to treat X-linked severe combined immunodeficiency (2018) (26)
Preliminary Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia (2019) (25)
Children are not little adults: just ask their hematopoietic stem cells. (2006) (24)
Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown for Sickle Cell Gene Therapy (2020) (24)
Small pituitary size in children with Fanconi Anemia (2007) (24)
The Changing Face of Adrenoleukodystrophy. (2020) (24)
Mathematical modeling of erythrocyte chimerism informs genetic intervention strategies for sickle cell disease (2016) (24)
Oxandrolone for the treatment of bone marrow failure in Fanconi anemia (2014) (24)
p21-activated kinase 2 regulates HSPC cytoskeleton, migration, and homing via CDC42 activation and interaction with β-Pix. (2016) (23)
Rac signaling in osteoblastic cells is required for normal bone development but is dispensable for hematopoietic development. (2012) (23)
HOXB4 Inhibits Cell Growth in a Dose-Dependent Manner and Sensitizes Cells Towards Extrinsic Cues (2006) (23)
Gene therapy continues to mature and to face challenges. (2009) (22)
Loss of the Rho GTPase activating protein p190-B enhances hematopoietic stem cell engraftment potential. (2007) (22)
Pediatric aplastic anemia and refractory cytopenia: A retrospective analysis assessing outcomes and histomorphologic predictors (2015) (21)
Broadening the indications for hematopoietic stem cell genetic therapies. (2013) (20)
Stem cell mobilization by β2-agonists (2006) (19)
Infantile Myelofibrosis and Myeloproliferation with CDC42 Dysfunction (2020) (19)
Induced pluripotent stem cells as a tool for gaining new insights into Fanconi anemia (2012) (19)
Validation of BCL11A As Therapeutic Target in Sickle Cell Disease: Results from the Adult Cohort of a Pilot/Feasibility Gene Therapy Trial Inducing Sustained Expression of Fetal Hemoglobin Using Post-Transcriptional Gene Silencing (2019) (19)
Clonal hematopoiesis in sickle cell disease (2021) (18)
A new mechanism of leukemia drug resistance? (2007) (18)
Cytokines secreted by bone marrow stromal cells protect c-KIT mutant AML cells from c-KIT inhibitor-induced apoptosis (2014) (18)
Contributions of the RhoGEF activity of p210 BCR/ABL to disease progression (2012) (18)
Gene therapy in the treatment of Fanconi anemia, a progressive bone marrow failure syndrome. (2005) (17)
Retroviral‐Fibronectin Interactions in Transduction of Mammalian Cells (1999) (17)
FIP1L1/PDGFRalpha synergizes with SCF to induce systemic mastocytosis in a murine model of chronic eosinophilic leukemia/hypereosinophilic syndrome. (2008) (17)
Stable differentiation and clonality of murine long-term hematopoiesis after extended reduced-intensity selection for MGMT P140K transgene expression. (2007) (16)
Flipping the Switch: Initial Results of Genetic Targeting of the Fetal to Adult Globin Switch in Sickle Cell Patients (2018) (15)
Comparative effects of in vivo treatment using interleukin-11 and stem cell factor on reconstitution in mice after bone marrow transplantation (1993) (15)
Foamy virus vectors come of age. (2008) (14)
Rapid lentiviral transduction preserves the engraftment potential of Fanca(-/-) hematopoietic stem cells. (2008) (14)
Outcome of Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome (2019) (14)
Leukemic survival factor SALL4 contributes to defective DNA damage repair (2016) (13)
Alpharetroviral self-inactivating vectors produced by a superinfection-resistant stable packaging cell line allow genetic modification of primary human T lymphocytes. (2016) (13)
Cutaneous infection with Metarhizium anisopliae in a patient with hypohidrotic ectodermal dysplasia and immune deficiency. (2008) (13)
In vivo enrichment of cytidine deaminase gene-modified hematopoietic cells by prolonged cytosine-arabinoside application. (2012) (12)
Stem cell mobilization by beta2-agonists. (2006) (12)
Charting a clear path: the ASGCT Standardized Pathways Conference. (2014) (12)
Gene Therapy Using a Self-Inactivating Lentiviral Vector Improves Clinical and Laboratory Manifestations of Wiskott-Aldrich Syndrome (2015) (11)
A recombinant human fibronectin fragment facilitates retroviral mediated gene transfer into human hematopoietic progenitor cells (1997) (11)
Real-Time PCR: an Effective Tool for Measuring Transduction Efficiency in Human Hematopoietic Progenitor Cells. (2005) (11)
HACE1 Prevents Lung Carcinogenesis via Inhibition of RAC-Family GTPases (2020) (11)
ESCGT 2008: progress in clinical gene therapy. (2009) (11)
Gene Transfer into Murine Hematopoietic Stem Cells and Bone Marrow Stromal Cells (1990) (9)
Perivascular deletion of murine Rac reverses the ratio of marrow arterioles and sinusoid vessels and alters hematopoiesis in vivo. (2015) (9)
The Rac 2 Guanosine Triphosphatase Regulates B Lymphocyte Antigen Receptor Responses and Chemotaxis and Is Required for Establishment of B-1 a and Marginal Zone B Lymphocytes 1 (2002) (9)
Current and future gene therapies for hemoglobinopathies. (2020) (9)
Lenti in red: progress in gene therapy for human hemoglobinopathies (2004) (9)
A Unique Carboxyl-terminal Insert Domain in the Hematopoietic-specific, GTPase-deficient Rho GTPase RhoH Regulates Post-translational Processing* (2013) (9)
RhoH participates in a multi-protein complex with the zinc finger protein kaiso that regulates both cytoskeletal structures and chemokine-induced T cells (2018) (9)
Lenti in red: progress in gene therapy for human hemoglobinopathies. (2004) (9)
The use of umbilical cord blood as a cellular source for correction of genetic diseases affecting the hematopoietic system (1995) (9)
Out of harm's way (2011) (8)
Restored macrophage function ameliorates disease pathophysiology in a mouse model for IL10 receptor deficient very early onset inflammatory bowel disease. (2021) (8)
Molecular Analysis of the Hematopoietic Microenvironment (1994) (8)
Updated Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia (2021) (7)
Retroviral transduction of murine and human hematopoietic progenitors and stem cells. (2014) (7)
Gene therapy needs both trials and new strategies (2004) (7)
CXCL 9 inhibits eosinophil responses by a CCR 3-and Rac 2-dependent mechanism (2005) (7)
Curing genetic disease with gene therapy. (2014) (6)
Role of p 38 and ERK MAP kinase in proliferation of erythroid progenitors in response to stimulation by soluble and membrane isoforms of stem cell factor (2002) (6)
The gp130 Cytokine Interleukin‐11 Regulates Engraftment of Vav1–/– Hematopoietic Stem and Progenitor Cells in Lethally Irradiated Recipients (2018) (6)
Evidence generation and reproducibility in cell and gene therapy research: A call to action (2021) (6)
Absence of the Rho GTPase Activating Protein p190-B Enhances Long Term Hematopoietic Stem Cell Engraftment (2008) (6)
Rac1/2 activation promotes FGFR1 driven leukemogenesis in stem cell leukemia/lymphoma syndrome (2020) (5)
Enhancing research in regenerative medicine. (2010) (5)
Transatlantic consortium spotlights need for changes in gene therapy trials. (2010) (5)
New AAV serotypes may broaden the therapeutic pipeline to human gene therapy. (2006) (5)
Pairing JAK with MEK for improved therapeutic efficiency in myeloproliferative disorders. (2019) (5)
Rac 1 is essential for intraembryonic hematopoiesis and for the initial seeding of fetal liver with definitive hematopoietic progenitor cells (2008) (5)
Gene therapy advances but struggles to interpret safety data in small animal models. (2006) (4)
Somatic Gene Therapy for X-Linked Severe Combined Immunodeficiency Using a Self-Inactivating Modified Gammaretroviral Vector Results in An Improved Preclinical Safety Profile and Early Clinical Efficacy in a Human Patient (2011) (4)
DROSHA Knockout Leads to Enhancement of Viral Titers for Vectors Encoding miRNA-Adapted shRNAs (2018) (4)
NIH recombinant DNA Advisory Committee continues to ponder adverse event associated with AAV gene therapy trial. (2008) (4)
Investigational curative gene therapy approaches to sickle cell disease (2021) (4)
Enhancement of mouse hematopoietic stem/progenitor cell function via transient gene delivery using integration-deficient lentiviral vectors (2018) (4)
Safety of Autologous Hematopoietic Stem Cell Transplantation with Gene Addition Therapy for Transfusion-Dependent β-Thalassemia, Sickle Cell Disease, and Cerebral Adrenoleukodystrophy (2020) (4)
Recombinant DNA advisory committee updates recommendations on gene transfer for x-linked severe combined immunodeficiency. (2009) (3)
Insertional Mutagenesis in Hematopoietic Cells: Lessons Learned from Adverse Events in Clinical Gene Therapy Trials (2011) (3)
Inhibition of the Rho GTPase, Rac, Differentially Inhibits Proliferation of AML Cell Lines and May Serve as a Novel Molecular Target. (2005) (3)
Chemotherapy-Only Preparative Regimen for Alternative Donor Hematopoietic Cell Transplantation for Patients with Fanconi Anemia (FA): Results of a Multi-Institutional Study (2015) (3)
Chemoprotective Gene Delivery (2008) (3)
Genetic deletion of Cdc 42 GAP reveals a role of Cdc 42 in erythropoiesis (2005) (3)
Optimization of Bcl11a Knockdown By miRNA Scaffold Embedded Shrnas Leading to Enhanced Induction of Fetal Hemoglobin in Erythroid Cells for the Treatment of Beta-Hemoglobinopathies (2014) (3)
Full Donor Myeloid Engraftment with Minimal Toxicity in Dyskeratosis Congenita Patients Undergoing Allogeneic Bone Marrow Transplantation without Radiation or Alkylating Agents (2014) (3)
Clonal Tracking By Whole Genome Sequencing Permits Comprehensive Mapping of the Genomic Landscape in Pre- and Post-Gene Therapy Sickle Cell Patients (2021) (3)
Chemotherapy-Only Preparative Regimen for T-Cell Depleted Hematopoietic Stem Cell Transplantation of Patients with Fanconi Anemia From Alternative Donors (2012) (3)
Rac1 and Rac2 GTPases Play Distinct Roles and Are Essential for Full Osteoclast Differentiation. (2005) (3)
Rapid development of pluripotent stem cells as a potential therapeutic modality. (2009) (3)
Hematopoietic SIN Lentiviral Micro RNA-Mediated Silencing of BCL11A: Pre-Clinical Evidence for a Sickle Cell Disease Gene-Therapy Trial (2012) (3)
1 Rac GTPase isoforms , Rac 1 and Rac 2 , play a redundant and crucial role in T-cell development Running title : Rac 1 and Rac 2 in T-cell development (2008) (3)
Adventitious mutations in clinical grade vectors: an issue to consider? (2006) (3)
National Institutes of Health releases new guidelines on human stem cell research. (2009) (3)
A rare complex DNA rearrangement in the murine Steel gene results in exon duplication and a lethal phenotype. (2003) (3)
FIP 1 L 1 / PDGFR α synergizes with SCF to induce systemic mastocytosis in a murine model of chronic eosinophilic leukemia / hypereosinophilic syndrome Running title : FIP 1 L 1-PDGFRA-induced systemic mastocytosis (2008) (3)
GATA2 Mutations In Pediatric Myelodysplastic Syndromes and Bone Marrow Failure (2013) (3)
Targeting Rac2 - Mitochondrial Respiratory Chain Complex III Signaling to Prevent Genomic Instability in Leukemia Stem and Progenitor Cells (2011) (2)
Rac1 and Rac2 Rho GTPases Distinctly Regulate Stem Cell Engraftment and Mobilization and Are Novel Targets for Pharmacologically-Induced Progenitor Mobilization. (2004) (2)
Gaining the hard yard: pre-clinical evaluation of lentiviral-mediated gene therapy for the treatment of β-thalassemia (2010) (2)
ASGT advises NIH on funding of gene therapy trials. (2007) (2)
A Call to Action on Sickle Cell Disease (2015) (2)
Deficiency of Rac1 and Rac2 GTPases Perturbs Erythroid Proliferation and Differentiation but Not Enucleation. (2006) (2)
An international conversation on Stem Cell Gene Therapy. 4th Stem Cell Conference on Stem Cell Gene Therapy, Thessaloniki, Greece, 13-17 September 2007. (2007) (2)
NIH funding of gene therapy trials. (2006) (2)
The Sooner the Better: Rapid Transduction Enhances the Engraftment/Selection of Gene Corrected Fanconi Anemia HSC. (2007) (2)
Long-Term In Vivo Expression of a Murine Adenosine Deaminase Gene in Rhesus Monkey Hematopoietic Cells of Multiple Lineages After Retroviral Mediated Gene Transfer Into CD 34 + Bone Marrow Cells (2003) (2)
Clinical Application of Gene Transfer Technology: Overcoming the Obstacles (2005) (2)
Aberrant overexpression of CD 14 on granulocytes sensitizes the innate immune response in mDia 1 heterozygous del ( 5 q ) MDS (2014) (2)
Methods for Gene Transfer: Genetic Manipulation of Hematopoietic Stem Cells (2007) (2)
Highly Efficient Therapeutic Gene Editing of BCL11A enhancer in Human Hematopoietic Stem Cells from ß-Hemoglobinopathy Patients for Fetal Hemoglobin Induction (2018) (2)
NIH decides against continuing NGVLs in their current form. (2007) (2)
Cross-talk between a 4 b 1 / a 5 b 1 and c-Kit results in opposing effect on growth and survival of hematopoietic cells via the activation of focal adhesion kinase , mitogen-activated protein kinase , and Akt signaling pathways (2001) (2)
IL-5 and the FIP1L1/PDGFRA Fusion Gene Cooperatively Promote Eosinophil Development, Activation, and Signal Transduction (2007) (2)
Unexpected help: mTOR meets lentiviral vectors. (2014) (2)
An emerging consensus on recommendations to facilitate clinical gene transfer. (2006) (2)
Stress-induced exit from dormancy alters redox signaling in HSCs, resulting in de novo DNA damage and bone marrow failure in the absence of a functional fanconi anemia signaling pathway (2014) (2)
Peripheral blood smears of children with multisystem inflammatory syndrome demonstrate prominence of early myeloid forms with morphologic evidence of toxic change (2020) (2)
Progress reported in two studies of clinical gene transfer into the retina. (2008) (2)
Deletion of Murine Rhoh induces More Aggressive Diffuse Large B Cell Lymphoma (DLBCL) Via Interaction with Kaiso and Regulation of BCL-6 Expression (2018) (2)
Interim Results from a Phase 2/3 Study of the Efficacy and Safety of Ex Vivo Gene Therapy With Lentiviral Vector (Lenti-D) for Childhood Cerebral Adrenoleukodystrophy (PL02.002) (2016) (2)
Cdc 42 Critically Regulates the Balance Between Myelopoiesis and Erythropoiesis Running title : Cdc 42 in myelopoiesis and erythropoiesis (2007) (2)
New approaches in the potential treatment of HIV-acquired immunodeficiency disease. (2009) (2)
AAV and Insertional Mutagenesis. (2007) (2)
Gene Therapy for Cerebral Adrenoleukodystrophy. (2018) (2)
Hematopoietic Cell Transplantation without Radiation or DNA Alkylating Agents in Bone Marrow Failure with Short Telomeres (2019) (2)
Immune Reconstitution and Preliminary Safety Analysis Of 9 Patients Treated With Somatic Gene Therapy For X-Linked Severe Combined Immunodeficiency (SCID-X1) With a Self-Inactivating Gammaretroviral Vector (2013) (2)
A Phase 2/3 Study of the Efficacy and Safety of Ex Vivo Gene Therapy with Lenti-DTM Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (2016) (2)
Chemotherapy-Only Preparative Regimen for Alternative Donor Hematopoietic Stem Cell Transplantation for Patients with Fanconi Anemia (2012) (1)
Comparison of Outcomes in Patients with Cerebral Adrenoleukodystrophy (CALD) Receiving Elivaldogene Autotemcel (eli-cel; Lenti-D) Gene Therapy in Clinical Trials Versus Those Receiving Allogeneic Hematopoietic Stem Cell Transplant in a Contemporaneous Comparator Study (2022) (1)
Pathways Formation through Multiple Distinct Effector Superoxide Production , and Myeloid Colony Rac 2 Regulates Neutrophil Chemotaxis (2005) (1)
An International Conversation on Stem Cell Gene Therapy: 4th Stem Cell Conference on Stem Cell Gene Therapy Thessaloniki, Greece, 13-17 September 2007. (2007) (1)
Pulmozyme® Treatment of Vectors Produced by Transient Transfection Reduces Residual Plasmid DNA on Human CD34+ Hematopoietic Progenitor Cells without Loss of Transduction or Engraftment Efficiency (2008) (1)
Interleukin-10 Signaling in Hematopoietic Stem and Progenitor Cells Maintains Stem Cell Function and Regulates Inflammation-Induced Myeloid Cell Output (2018) (1)
Updated Safety and Efficacy Outcomes from an Ongoing Phase 2/3 Trial of Lenti-D Autologous Hematopoietic Stem Cell Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (2019) (1)
O-025 GATA2 mutations in pediatric myelodysplastic syndrome and bone marrow failure disorders (2013) (1)
Rac1 Regulates Migration of Yolk Sac Hematopoietic Progenitors into the Blood, a Process Essential for Subsequent Seeding of Fetal Liver Hematopoiesis. (2005) (1)
Effects of BCL11A Shmir-Induced Post-Transcriptional Silencing on Hemoglobin Polymer Inhibition in Single Red Blood Cells at Physiologic Oxygen Tension (2021) (1)
Of Mouse Models and Men. (2007) (1)
Regulation of Rho GTPases by the Hematopoietic-Specific Guanine Nucleotide Exchange Factor Vav1 Is Critical for Hematopoietic Stem Cell Retention in the Endosteal Niche and Engraftment. (2009) (1)
Ectopic HOXB 4 overcomes the inhibitory effect of tumor necrosis factor-on Fanconi anemia hematopoietic stem and progenitor cells (2009) (1)
In Vivo Bone Marrow Stem Cell Gene Transfer in Mice by In Situ Delivery of a 3rd-Generation Lentiviral Vector Using Intrafemoral Injection. (2004) (1)
In Vivo Bone Marrow Stem Cell Gene Transfer in Mice by In Situ Delivery of a 3rd-Generation Lentiviral Vector Using Intrafemoral Injection. (2004) (1)
Congenital X‐linked neutropenia with myelodysplasia and somatic tetraploidy due to a germline mutation in SEPT6 (2021) (1)
Intact Rac Signaling Is Important for Leukemia Cell Survival (2010) (1)
Cdc42 Is Critical for Multi-Lineage Blood Cell Development from Hematopoietic Stem and Progenitor Cells. (2006) (1)
The Role of Rac and Pak in Neutrophil Histone Hypercitrullination and Neutrophil Extracellular Traps Formation (2014) (1)
Erythrocyte Cytoskeletal Defects Induced in Mice by Deletion of Rac GTPases. (2004) (1)
Diabetic / Severe Combined Immunodeficiency ( NOD / SCID ) Mouse as a Model System to Study the Engraftment and Mobilization of Human Peripheral Blood Stem Cells (1998) (1)
Rac GTPases Regulate Erythropoiesis Both in the Early Steps of Differentiation and in Enucleation. (2007) (1)
Defining Current Strategies for Future Human Trials in Gene Therapy (2000) (1)
A Critical Role of the Rho Family GTPase Cdc42 in Hematopoietic Stem Cell Mobilization, Homing, Engraftment and Differentiation. (2005) (1)
The RHO GTPASE CDC42 Regulator CDC42GAP Plays a Critical Role in Neutrophil Migration Via Podosome-Like Formation. (2004) (1)
Genetically modified hematopoietic stem cells expressing o6methylguanine dna methyltransferase via a retrovirus vector protect the lymphoid and stem cell compartments in long-term engrafted animals treated with nitrosourea (1996) (1)
Outcomes of Immunosuppressive Therapy for Pediatric Aplastic Anemia: A North American Pediatric Aplastic Anemia Consortium (NAPAAC) Study (2017) (1)
The Rho GTPase Rac1 Is Not Required for Definitive Hematopoietic Specification in ES-Derived Hematopoiesis. (2009) (1)
HEMATOPOIESIS AND STEM CELLS p21-activated kinase 2 regulates HSPC cytoskeleton, migration, and homing via CDC42 activation and interaction with b -Pix (2016) (1)
Gene-Correction Rescues Reprogramming of Fanconi Anemia Fibroblasts and Enables Hematopoietic Differentiation of FA Induced Pluripotent Stem Cells in Vitro and In Vivo (2011) (1)
Elivaldogene Autotemcel (eli-cel, Lenti-D) Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy: Updated Results from the Phase 2/3 ALD-102 Study and First Report on Safety Outcomes from the Phase 3 ALD-104 Study (2021) (1)
Rac GTPase Apoptotic Cells by Modulating Activation of PTEN Negatively Regulates Engulfment of (2011) (0)
Lenti-D Hematopoietic Stem Cell Gene Therapy to Arrest Progression of Cerebral Adrenoleukodystrophy: Interim Results of an International Phase 2/3 Trial (2018) (0)
Human Hematopoietic Stem Cells, Progenitors, and Peripheral Blood Lymphocytes as Targets for the Correction of Immune System Disorders via Gene Therapy (1998) (0)
P190-B RhoGAP Is Critical for Hematopoietic Stem/Progenitor Cell Homing and Short Term Engraftment. (2006) (0)
Too little, too late: The initial experience in FA gene therapy (2008) (0)
Regulation of the Rho GTPase, RhoA, Activity Is Critical for Erythropoiesis during Embryo Development. (2005) (0)
Rho GTPase Activating Protein p190-B Regulates Hematopoietic Stem Cell Fate Decision (2010) (0)
A Prospective Trial of Minimal Intensity Conditioning with Fludarabine and Anti-CD52 Antibody Alone in Dyskeratosis Congenita (2014) (0)
Group A Pak Proteins Are Essential for HSC/P Engraftment. (2010) (0)
Self-inactivating retroviral vectors for gene therapy of SCID-X1 (2008) (0)
Congenital X-Linked Myelodysplasia with Tetraploidy Is Associated with De Novo Germline C-Terminal Mutation of SEPT6, a Septin Filament Protein (2018) (0)
Vav3 and Rac2 Activation Are Required for B Progenitor Transformation Induced by p190-BCR-ABL. (2009) (0)
Human Peripheral Blood Stem Cells Mouse as a Model System to Study the Engraftment and Mobilization of Nonobese Diabetic/Severe Combined Immunodeficiency (NOD/SCID) (2013) (0)
Deletion of murine Rhoh leads to de-repression of Bcl-6 via decreased KAISO levels and accelerates a malignancy phenotype in a murine model of lymphoma (2022) (0)
Small Molecule Inhibition of PDE6D-RAS Interaction Suppresses the Growth of Acute Lymphoblastic Leukemia Cell Lines (2021) (0)
Enough is indeed enough: ACGME required changes in pediatric training (2012) (0)
Rac GTPase Survival Signaling Through Anti-Apoptotic Bcl-2 Proteins Reveals a Target for Combination Therapy in MLL-AF9 Acute Myeloid Leukemia (2011) (0)
Kinase Activity Of Pak2, An Effector Of Rac/CDC42 and Its Interaction With β-PIX Is Required For Murine Hematopoietic Stem Cell Shape, F-Actin Formation, Directional Migration In Vitro and For HSPC Homing To Bone Marrow In Vivo (2013) (0)
Rac2 biologic functions and intracellular localization The TRQQKRP motif located near the C-terminus of Rac2 is essential for (2013) (0)
Mechanisms of Resistance to Reprogramming of Cells Defective In the Fanconi Anemia DNA Repair Pathway (2010) (0)
Functional Characterization of the RhoH c-Terminus and Insert Domain As Potential Drug Targets (2011) (0)
314. Mathematical Modeling of Erythrocyte Chimerism Informs Clinical Strategies for Sickle Cell Disease (2016) (0)
HSC Exit From Dormancy Provokes De Novo DNA Damage, Leading To Bone Marrow Failure If Unresolved By The Fanconi Anemia Pathway (2013) (0)
Correction and apology. (2009) (0)
A pox on your tumor. (2006) (0)
The Expression of the GTPase-Deficient, Hematopoietic-Specific RhoH GTPase Is Implicated in Development of Chronic Lymphocytic Leukemia (CLL). (2007) (0)
The long road traveled in hematopoietic stem cell gene therapy. (2022) (0)
The NIH roadmap: timing is everything. (2005) (0)
Role of rac 2 in hemopoietic stem cell motility (2000) (0)
Health Centers : Navigating the “ Roadmap ” (2007) (0)
Rac2 GTPase Activation Is Necessary for Development of p190-BCR-ABL-Induced B-Cell Acute Lymphoblastic Leukemia (2008) (0)
Effects of BCL11A Shmir-Induced Post-Transcriptional Silencing on Distributions of HbF in Single-RBCs and Reticulocytes (2021) (0)
Vav1 Regulates Perivascular Homing, Bone Marrow Retention and Engraftment of Hematopoietic Stem Cells Via SDF1a Signaling (2010) (0)
Gene transfer and gene therapy (2015) (0)
Rhogtpase RAC2 Is Required for In Vivo Transformation Activity by P210 Bcr-Abl Fusion Oncogene. (2004) (0)
EXPRESSION of ACTIVATED of MAP KINASE KINASE IS SUFFICIENT for MEGAKARYOCYTIC DIFFERENTIATION of CMK CELLS (1996) (0)
proteins as combination targeted therapy in MLL-AF9 leukemia Inhibition of Rac GTPase signaling and downstream prosurvival Bcl-2 (2011) (0)
Chemoprotection of Hematopoietic Stem Cells (2008) (0)
T Cell Lymphopenia in Rhoh−/− Mice Results from Combined Defects of T Cell Migration and IL-7-Regulated Naïve T Cell Homeostasis. (2006) (0)
SAFETY AND EFFICACY OF GENE THERAPY USING A MODIFIED SELF-INACTIVATING GAMMARETROVIRAL VECTOR FOR SCID-X1 (2017) (0)
Pediatric Aplastic Anemia and Refractory Cytopenia of Childhood: A Retrospective Single Institutional Analysis to Determine Outcomes and Histomorphological Predictors (2014) (0)
BPX-501 Cells (Donor T Cells Transduced with iC9 Suicide Gene) Treatment Following TCR Alpha Beta Depleted Stem Cell Transplantation in Adults and Children with Hematological Disorders (2016) (0)
Alteration of RhoH Expression Is Associated with Human Diffuse Large B-Cell Lymphoma. (2004) (0)
Progress in genetic therapy for severe combined immunodeficiency associated with adenosine deaminase deficiency. (2009) (0)
Cross-Talk between Rho GTPases Regulates Actin Cytoskeleton and Chemotaxis of Hematopoietic Progenitor Cell. (2005) (0)
FIP1L1/PDGFRA (F/P) Fusion Gene Induces Eosinophil Development, Activation, and Survival Synergistically with SCF and IL-5 (2008) (0)
Leukemia Stem Cells Are Resistant to In Vivo, Cell Non-Autonomous Wnt Inhibition. (2009) (0)
Retroviral infection on fibronectin: Preclinical studies (1998) (0)
Investigation of Cell-Contact Dependent Alterations in Immune Synapse Formation between T Cells and BCP-ALL Cells (2019) (0)
A "vector drain" in US gene therapy development? (2008) (0)
SCF and IL-5 Synergize with FIP1L1/PDGFRα To Induce Mastocytosis in a Chronic Eosinophilic Leukemia Murine Model. (2006) (0)
Gene Therapy/Editing (2016) (0)
The hematopoietic-specific rho gtpase, RAC2, is essential for normal adhesion and controlled movement of hematopoietic progenitor cells In vitro And In vivo (2000) (0)
SYNGAP1 Developmental and Epileptic Encephalopathy: Delineating the Phenotypic Spectrum (2018) (0)
Xeroderma Pigmentosum Group B Binding Contributes to p210 BCR/ABL Mediated Transformation in CML. (2008) (0)
Very High Expression of MGMT(P140K) Paradoxically Impairs Proliferation, In Vivo Selection and Protection of Transduced Hematopoietic Stem Cells. (2007) (0)
Differentiation Kinase-Mediated Mitogen Activated Protein Kinase Activation in Cell and Thymocyte Development: A Role for Receptor Tyrosine Overexpression of Human Stem Cell Factor Impairs Melanocyte, Mast Cell, (2013) (0)
Enhancing Research in Regenerative Medicine (2011) (0)
Increased, mediated by virus-dna-transfer (1995) (0)
Effective Lentiviral Gene Therapy for X-Linked Chronic Granulomatous Disease (X-CGD) (2019) (0)
erythrocyte cytoskeleton Rac GTPases regulate the morphology and deformability of the (2013) (0)
Cells and Genes in Molecular Therapeutics (2000) (0)
Earnest Support for Careers and Cures (2015) (0)
Improved Engraftment of Bone Marrow and Mobilized Peripheral Blood Stem Cells in a Fanconi Anemia Murine Model. (2006) (0)
erythropoiesis Cdc42 critically regulates the balance between myelopoiesis and (2013) (0)
Leukemia stem cells occupy niches that are physically distinct and independent of the normal constraints that apply to hematopoietic stem cells (2010) (0)
829. Design of a Safe and Efficient Vector for Bone Marrow Chemoprotection by O6-Methyl-Guanine-DNA-MethylTransferase (2005) (0)
Vav1, a Rho GTPase Guanine Exchange Factor (GEF) Is Required to Integrate gp130 Cytokine Signaling for Successful Engraftment of Murine Hematopoietic Stem Cells Following Irradiation (2016) (0)
459. Murine Study To Evaluate Long-Term Transgene Expression of the MSCV-MGMTP140K wc Retroviral Vector in Support of a Phase I Gene Transfer Trial-Limitations of the Murine Model as a Pre-Clinical Tool (2006) (0)
Early Results of the Clinical Collection and Genetic Correction of Fanconi Complement Type A Hematopoietic Stem Cells. (2005) (0)
282. Circumventing Hematopoietic Toxicities and Reversing Sickle Phenotype by Lineage-Specific BCL11A Knock Down and γ-Globin Induction (2016) (0)
Managing childhood cancer survivors at risk for stroke: Practice norms and areas of controversy. (2018) (0)
The RhoH GTPase Interacts with ZAP-70 Kinase and Is Required for T Cell Receptor Signaling and T Cell Development. (2005) (0)
FDA guidance document on monitoring delayed adverse events a good first start. (2005) (0)
The Ras-related Rho GTPases, Rac1 and Rac2 regulate the homing, engraftment and mobilization of hematopoietic stem cells and progenitor cells (2003) (0)
97. Translational Studies toward Optimizing In Vivo Bone Marrow Stem Cell Gene Transfer Using Lentiviral Vector (2006) (0)
Making Sense of New Drug Discovery (2015) (0)
Production of High Titer cGMP-Grade SIN Gamma-Retroviral Vectors by Transfection in a Closed System Bioreactor (2008) (0)
6. In Vivo Adult Stem Cell Gene Transfer in Mice by In Situ Delivery of a Self-Inactivating Lentiviral Vector Using Intrafemoral Injection (2005) (0)
Pak A proteins are essential for hematopoietic stem/progenitor cell (HSC/P) engraftment through regulation of signaling pathways controlling HSC/P proliferation, survival, migration, and the actin cytoskeleton (2011) (0)
A Phase 2, Open-Label Study to Evaluate the Efficacy and Safety of Mgta-145 in Combination with Plerixafor for the Mobilization of Hematopoietic Stem Cells in Patients with Sickle Cell Anemia (2022) (0)
682. Collection and Genetic Correction of Fanconi Complement Type A Hematopoietic Stem Cells: Results of Two Pilot Studies (2006) (0)
ASGCT 20(th) Anniversary Special Issue of Molecular Therapy: Evolving Gene Therapy in Primary Immunodeficiency (2017) (0)
Deletion of murine Rac gtpases in the perivascular niche disrupts trabecular bone formation, medullary blood vessels and hematopoiesis (2013) (0)
ASH as a Global Organization (2015) (0)
Inhibition Of Telomerase Is a Novel and Effective Therapy In MLL-Rearranged Acute Myeloid Leukemia (AML) (2013) (0)
Lenti-D Hematopoietic Stem Cell Gene Therapy Stabilizes Neurologic Function in Boys with Cerebral Adrenoleukodystrophy (2020) (0)
Leading the Charge for Precision Medicine (2015) (0)
Author Feedback Program Reveals Satisfaction with the Review Process at Molecular Therapy (2005) (0)
Rac2 Function in Eosinophil Superoxide Generation and Allergic Airway Inflammation (2008) (0)
Vector insertion, mutagenesis and transgene toxicity. (2006) (0)
Rac1 and Rac2 GTPases Play Redundant but Critical Role in T-Cell Development and Survival. (2005) (0)
Vav1 Deficiency Differentially Affects Engraftment of Fetal Hematopoietic Organs During Development Compared with Adult Marrow After Transplantation (2012) (0)
The FIP1-like-1 (FIP1L1)- Platelet-Derived Growth Factor Receptor Alpha (PDGFR alpha) fusion gene cooperates with IL-5 to induce a hypereosinophilic syndrome (HES)-like disease in a murine model (2005) (0)
Bcl-XL Is a Critical Mediator of Rac Signaling in MLL-AF9-Induced Acute Myeloid Leukemia. (2009) (0)
Two Decades of Molecular Therapy and a 35-Year Personal View of Changes in Gene Therapy. (2019) (0)
Cooperation of KITD816V and TET2 loss of function in the pathogenesis of systemic mastocytosis and associated hematological malignancies (2013) (0)
engraftment and hematopoietic stem/progenitor cell survival, adhesion, and Genetic deletion of Cdc42GAP reveals a role of Cdc42 in erythropoiesis (2014) (0)
Rac GTPases Are Required for MLL-AF9-Induced Mixed Lineage Leukemia. (2008) (0)
The Rho Family GTPase Cdc42 Regulates Multiple Hematopoietic Stem/Progenitor Cell Functions and Erythropoiesis. (2004) (0)
Rac2 GTPase Plays a Critical Role in Platelet Adhesion as Well as in Sustenance and Perpetuation of Platelet Aggregation. (2004) (0)
in Vivo and in Vitro Nitrosourea-induced Toxicity Bone Marrow Protects Hematopoietic Cells from Retrovirus-mediated Expression of a DNA Repair Protein in Updated (2006) (0)
Gfi1 as a Regulator of Hematopoietic Differentiation. (2008) (0)
progenitor cell proliferation and engraftment Inhibition of RhoA GTPase activity enhances hematopoietic stem and (2013) (0)
Are lentivirus vectors safer? (2007) (0)
Aberrant Overexpression Of CD14 In Granulocytes Sensitizes Innate Immune Response In mDia1 Heterozygous Myelodysplastic Syndromes (2013) (0)
Combined +58 and +55 BCL11A enhancer Editing Yields Exceptional Efficiency, Specificity and HbF Induction in Human and NHP Preclinical Models (2021) (0)
A Cell-Autonomous Defect of Bone Marrow Hematopoietic Stem/Progenitor Cells and Impaired Stromal Function in FA-a Patients (2008) (0)
DNA transfer mediated improved virus. (1995) (0)
Ectopic HOXB4 Overcomes the Inhibitory Effect of TNFα on Hematopoietic Stem and Progenitor Cells in a Murine Fanconi Anemia Model. (2008) (0)
Hematology grants workshop. (2010) (0)
FIP1L1-PDGFRα Induces Stem Cell Factor Dependent Eosinophil and Mast Cell Development from Murine Hematopoietic Stem Cell/Progenitors in Vitro (2006) (0)
Investigation of T Cell Subsets and Germinal Center Reaction in a B Cell Malignancy Model of Mice Deficient in the Lymphoma Hyermutable Gene Rhoh (2014) (0)
Rac2, is associated with a human phagocyte immunodeficiency Dominant negative mutation of the hematopoietic-specific Rho GTPase, (2011) (0)
Stable Polyclonal Murine Long-Term Hematopoiesis without Clonal Exhaustion of MGMT P140K Expressing Murine Hematopoietic Stem Cells after Extended Reduced Intensity Selection. (2006) (0)
The Ski oncoprotein regulates Hematopoietic Stem Cell Fitness and Functions as a Corepressor for Gfi1. (2008) (0)
Molecular Therapy Moves on to a New Editor-in-Chief. (2009) (0)
Retrovirus-Mediated GeneTransfer ofHumanAdenosine Deaminase: Expression ofFunctional EnzymeinMurine Hematopoietic StemCells InVivo (1987) (0)
Too much of a good thing? MGMT in chemoresistance and brain tumor therapy (2008) (0)
2018 Karl Landsteiner Award (2018) (0)
Altered HSC Metabolism in Response to Stress Leads to De Novo dna Damage and Cellular Attrition (2014) (0)
RAC2 Regulates the activation of akt and the balance between pro- and anti-apoptotic proteins bad and bcl-xl (2000) (0)
RhoH-Deficient Mice Show Altered B Cell Populations In Vivo. (2007) (0)
migration in mast cells on class IA PI-3kinase and Rac pathway in regulating integrin directed 4 integrin-mediated signals α Genetic evidence for convergence of c-Kit and (2013) (0)
Padi4 Regulates NET Formation and Inflammatory Cell Death Downstream of Mlkl (2018) (0)
FIP1L1/PDGFRa Synergizes with SCF/c-Kit Signaling To Induce Systemic Mastocytosis in a Chronic Eosinophilic Leukemia Murine Model (2007) (0)
Rac GTPases in Human Diseases The Harvard community has made this article openly available. Please share how this access benefits you. Your story matters (2010) (0)
Creation of a Fully Functional Human Chimeric (1998) (0)
progenitors genomic instability in chronic myeloid leukemia stem cells and primitive mitochondrial respiratory chain complex III-generated ROS cause - Rac2 (2012) (0)
Long-term expression ofhumanadenosine deaminase inmice transplanted withretrovirus-infec (1989) (0)

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