Professor, Pediatrics, UCLA School of Medicine
Pediatrics
Dr. Donald B. Kohn, MD
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Doctor Donald B. Kohn, MD works in the field of Pediatrics. He went to medical school at University of Wisconsin School of Medicine and Public Health. He received awards:"Super Doctor". Donald Kohn has written publications. He has 97 publications published with PubMed. The lastest paper is: 'A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells.'
Publications
- A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells.
- Unrelated donor hematopoietic stem cell transplantation for the treatment of non-malignant genetic diseases: An alemtuzumab based regimen is associated with cure of cl...
- Gene therapy outpaces haplo for SCID-X1.
- Correction of the sickle-cell disease mutation in human hematopoietic stem/progenitor cells.
- Potentially therapeutic levels of anti-sickling globin gene expression following lentivirus-mediated gene transfer in sickle cell disease bone marrow CD34(+) cells.
- Rescue of splicing-mediated intron loss maximizes expression in lentiviral vectors containing the human ubiquitin C promoter.
- A Reduced-Toxicity Regimen Is Associated with Durable Engraftment and Clinical Cure of Nonmalignant Genetic Diseases among Children Undergoing Blood and Marrow Transpl...
- T-cell immunotherapy: looking forward.
- HSV-sr39TK positron emission tomography and suicide gene elimination of human hematopoietic stem cells and their progeny in humanized mice.
- Effects of Vector Backbone and Pseudotype on Lentiviral Vector-Mediated Gene Transfer: Studies in Infant ADA-Deficient Mice and Rhesus Monkeys.
- Human Lymphoid Development in the Absence of Common γ-Chain Receptor Signaling.
- HIV eradication-from Berlin to Boston.
- Gene Therapy: Charting a Future Course Summary of a National Institutes of Health Workshop, April 12, 2013.
- Dissecting the Mechanism of Histone Deacetylase Inhibitors to Enhance the Activity of Zinc Finger Nucleases Delivered by Integrase-Defective Lentiviral Vectors.
- Erythropoiesis from human embryonic stem cells through erythropoietin-independent AKT signaling.
- Hematopoietic stem cells for cancer immunotherapy.
- Establishing diagnostic criteria for severe combined immunodeficiency disease (SCID), leaky SCID, and Omenn syndrome: The Primary Immune Deficiency Treatment Consortiu...
- Pre-clinical demonstration of lentiviral vector mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency.
- Primary Immune Deficiency Treatment Consortium (PIDTC) report.
- Newborn screening for severe combined immunodeficiency and T-cell lymphopenia in California: Results of the first 2 years.
- The natural history of children with severe combined immunodeficiency: baseline features of the first fifty patients of the primary immune deficiency treatment consort...
- Integrase-defective Lentiviral Vectors as a Delivery Platform for Targeted Modification of Adenosine Deaminase Locus.
- Gene Therapy for Rare Diseases: Summary of a National Institutes of Health Workshop, September 13, 2012.
- Allelic Exclusion and Peripheral Reconstitution by TCR Transgenic T Cells Arising From Transduced Human Hematopoietic Stem/Progenitor Cells.
- Long-term in vivo monitoring of mouse and human hematopoietic stem cell engraftment with a human positron emission tomography reporter gene.
- Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans.
- Hematopoietic Stem Cell Gene Therapy for the Multisystemic Lysosomal Storage Disorder Cystinosis.
- Gene Therapy by Autologous Transplant of Gene-Modified HSC.
- Gene therapy/bone marrow transplant in ADA-deficient mice: roles of enzyme replacement therapy and cytoreduction.
- Novel pathways to erythropoiesis induced by dimerization of intracellular C-mpl in human hematopoietic progenitors.
- Antitumor activity from antigen-specific CD8 T cells generated in vivo from genetically engineered human hematopoietic stem cells.
- A Tale of Two SCIDs.
- Highly efficient large-scale lentiviral vector concentration by tandem tangential flow filtration.
- Myeloid dysplasia and bone marrow hypocellularity in adenosine deaminase-deficient severe combined immune deficiency.
- CARs on track in the clinic.
- Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo.
- Regulated expansion of human pancreatic beta-cells.
- Update on gene therapy for immunodeficiencies.
- Neurocognitive function of patients with severe combined immunodeficiency.
- Improving cellular therapy for primary immune deficiency diseases: recognition, diagnosis, and management.
- Stable transgene expression in primitive human CD34+ hematopoietic stem/progenitor cells, using the Sleeping Beauty transposon system.
- Preloading potential of retroviral vectors is packaging cell clone dependent and centrifugation onto CH-296 ensures highest transduction efficiency.
- Long-term neurocognitive function of pediatric patients with severe combined immune deficiency (SCID): pre- and post-hematopoietic stem cell transplant (HSCT).
- Gene therapy fulfilling its promise.
- Pre- and post-natal treatment of hemophagocytic lymphohistiocytosis.
- Allogeneic hematopoietic cell transplantation for primary immune deficiency diseases: current status and critical needs.
- CD4+CD25- T cells transduced to express MHC class I-restricted epitope-specific TCR synthesize Th1 cytokines and exhibit MHC class I-restricted cytolytic effector func...
- In vivo biosafety model to assess the risk of adverse events from retroviral and lentiviral vectors.
- Neonatal bone marrow transplantation of ADA-deficient SCID mice results in immunologic reconstitution despite low levels of engraftment and an absence of selective don...
- Progressive declines in neurocognitive function among survivors of hematopoietic stem cell transplantation for pediatric hematologic malignancies.
- Expansion of multipotent and lymphoid-committed human progenitors through intracellular dimerization of Mpl.
- Redirecting specificity of T-cell populations for CD19 using the Sleeping Beauty system.
- Human hematopoietic cell culture, transduction, and analyses.
- Foamy virus vectors expressing anti-HIV transgenes efficiently block HIV-1 replication.
- Genetic therapies against HIV.
- Manipulation of OCT4 levels in human embryonic stem cells results in induction of differential cell types.
- The effects of Campath 1H upon graft-versus-host disease, infection, relapse, and immune reconstitution in recipients of pediatric unrelated transplants.
- Prolonged pancytopenia in a gene therapy patient with ADA-deficient SCID and trisomy 8 mosaicism: a case report.
- Lentiviral vector transduction of a dominant-negative Rev gene into human CD34+ hematopoietic progenitor cells potently inhibits human immunodeficiency virus-1 replica...
- Effective suicide gene therapy for leukemia in a model of insertional oncogenesis in mice.
- Lentiviral vectors ready for prime-time.
- Association of prostate-specific membrane antigen with caveolin-1 and its caveolae-dependent internalization in microvascular endothelial cells: implications for targe...
- Transient gene expression by nonintegrating lentiviral vectors.
- In vivo transduction by intravenous injection of a lentiviral vector expressing human ADA into neonatal ADA gene knockout mice: a novel form of enzyme replacement ther...
- Tolerance induction by lentiviral gene therapy with a nonmyeloablative regimen.
- Specific and stable gene transfer to human embryonic stem cells using pseudotyped lentiviral vectors.
- Perspectives on gene therapy for immune deficiencies.
- Long-term efficacy of enzyme replacement therapy for adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID).
- Successful hematopoietic stem cell transplantation for Niemann-Pick disease type B.
- Intrapulmonary and intramyocardial gene transfer in rhesus monkeys (Macaca mulatta): safety and efficiency of HIV-1-derived lentiviral vectors for fetal gene delivery.
- Selective survival of peripheral blood lymphocytes in children with HIV-1 following delivery of an anti-HIV gene to bone marrow CD34(+) cells.
- Neonatal gene therapy of MPS I mice by intravenous injection of a lentiviral vector.
- Fetal gene transfer using lentiviral vectors and the potential for germ cell transduction in rhesus monkeys (Macaca mulatta).
- Factors influencing the titer and infectivity of lentiviral vectors.
- Integrated self-inactivating lentiviral vectors produce full-length genomic transcripts competent for encapsidation and integration.
- Retrovirally transduced bone marrow has a therapeutic effect on brain in the mouse model of mucopolysaccharidosis IIIB.
- Use of lentiviral vectors to induce long-term tolerance to gal(+) heart grafts.
- Simian immunodeficiency virus infection of hematopoietic stem cells and bone marrow stromal cells.
- Busulfan and cyclophosphamide as a conditioning regimen for pediatric acute lymphoblastic leukemia patients undergoing bone marrow transplantation.
- Morphological analysis and lentiviral transduction of fetal monkey bone marrow-derived mesenchymal stem cells.
- Dynamic tracking of human hematopoietic stem cell engraftment using in vivo bioluminescence imaging.
- Scaffold attachment region-containing retrovirus vectors improve long-term proviral expression after transplantation of GFP-modified CD34+ baboon repopulating cells.
- The Moloney murine leukemia virus repressor binding site represses expression in murine and human hematopoietic stem cells.
- Transduction of green fluorescent protein increased oxidative stress and enhanced sensitivity to cytotoxic drugs in neuroblastoma cell lines.
- Treatment of the mouse model of mucopolysaccharidosis I with retrovirally transduced bone marrow.
- Lentivirus vectors incorporating the immunoglobulin heavy chain enhancer and matrix attachment regions provide position-independent expression in B lymphocytes.
- Efficient characterization of retro-, lenti-, and foamyvector-transduced cell populations by high-accuracy insertion site sequencing.
- Effects of the negative control region on expression from retroviral LTR.
- Clonality analysis after retroviral-mediated gene transfer to CD34+ cells from the cord blood of ADA-deficient SCID neonates.
- Gene therapy of RAG-2-/- mice: sustained correction of the immunodeficiency.
- Marrow-derived cells as vehicles for delivery of gene therapy to pulmonary epithelium.
- Expression from second-generation feline immunodeficiency virus vectors is impaired in human hematopoietic cells.
- Kinetics of fluorescence expression in nonhuman primates transplanted with GFP retrovirus-modified CD34 cells.
- Adenosine deaminase gene therapy protocol revisited.
- Requirement for NK cells in CD40 ligand-mediated rejection of Philadelphia chromosome-positive acute lymphoblastic leukemia cells.
- Second hematopoietic stem cell transplantation in pediatric patients: overall survival and long-term follow-up.
Schools
University of Wisconsin Madison
University Of Wisconsin Madison
National Institute Of Health
Doctors Specialties
- Pediatrics
Accepted Insurances
- Aetna HMO
- Choice Plus POS II
- Blue Card PPO
- CaliforniaCare HMO
- Advantage Preferred DirectAccess Plus PPO
- Blue Cross Blue Shield of California PPO
- Pathway X Direct Access PPO
- Pathway X Tiered Direct Access
- Select Plus HMO
- Select PPO
- Cigna HMO
- Great West Healthcare-Cigna PPO
- Commercial Insurance Company
- Blue & Gold HMO
- Covered California - Bronze PPO
- Employer Group HMO
- Health Net PPO
- Individual and Family PPO
- Medicare MCR
- Molina Healthcare
- Kaiser PHCS Network PPO
- MultiPlan PPO
- PHCS Network PPO
- Heritage Signature
- Choice Plus POS
- Navigate HMO
- Navigate POS
- Options PPO
- PacifiCare HMO
Awards
- Super Doctor
Education
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National Institutes of Health Clinical Center -
University of Wisconsin -
University of Wisconsin School of Medicine and Public Health
Hospital
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Mattel Children's Hospital UCLA -
Ronald Reagan UCLA Medical Center -
Santa Monica-UCLA Medical Center and Orthopaedic Hospital
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