Gene Therapy Using Hematopoietic Stem Cells View Homepage


Ontology type: schema:MonetaryGrant     


Grant Info

YEARS

2003-2015

FUNDING AMOUNT

18258360.0 USD

ABSTRACT

DESCRIPTION (provided by applicant): The central theme of this Program is improving gene transfer and gene correction in human hematopoietic stem and progenitor cells to provide more effective and safe therapies for blood cell disorders. While the concept of gene therapy using HSC to provide improved methods to treat congenital disorders has been under study for at least two decades, there have been only a small number of successful clinical applications. The techniques currently in use for gene transfer and expression in HSC are sub-optimal in most cases to yield clinical benefits. The goals of this Program are to investigate the mechanisms limiting successful clinical applications of gene transfer and to develop improved techniques which will broaden the range of diseases which may be treated effectively and safely. These advances can only be realized by combining each of these individual projects into a unified Program. This Program has three projects: 1. Selective Expansion of Hematopoietic Stem Cells and Lymphoid Progenitors, 2. Genome Modification in Human Hematopoietic Stem cells, and 3. Gene Therapy for ADA-deficient SCID. Four Cores (Administrative, Cell Isolation and Analysis, Vectors and Animals) will support the projects with integrated services for optimal quality and efficiency. The Project and Core leaders have complementary expertise in the relevant areas of experimental hematology, gene therapy, immunology, and signal transduction and have a long-standing record of interactive collaborations. More... »

URL

http://projectreporter.nih.gov/project_info_description.cfm?aid=8513393

Related SciGraph Publications

  • 2017-08-25. Adenosine Deaminase (ADA)-Deficient Severe Combined Immune Deficiency (SCID): Molecular Pathogenesis and Clinical Manifestations in JOURNAL OF CLINICAL IMMUNOLOGY
  • 2017-04-03. Generation of mature T cells from human hematopoietic stem/progenitor cells in artificial thymic organoids in NATURE METHODS
  • 2017-02-24. The T-ALL related gene BCL11B regulates the initial stages of human T-cell differentiation in LEUKEMIA
  • 2016-05-16. Medial HOXA genes demarcate haematopoietic stem cell fate during human development in NATURE CELL BIOLOGY
  • 2015-12-22. BALR-6 regulates cell growth and cell survival in B-lymphoblastic leukemia in MOLECULAR CANCER
  • 2015-12-01. LncRNA profiling of human lymphoid progenitors reveals transcriptional divergence of B and T lineages in NATURE IMMUNOLOGY
  • 2015-11-09. Homology-driven genome editing in hematopoietic stem and progenitor cells using zinc finger nuclease mRNA and AAV6 donors in NATURE BIOTECHNOLOGY
  • 2014-04-08. HIV eradication—from Berlin to Boston in NATURE BIOTECHNOLOGY
  • 2013-01-29. A CD19/Fc fusion protein for detection of anti-CD19 chimeric antigen receptors in JOURNAL OF TRANSLATIONAL MEDICINE
  • 2012-12-30. Critical Differences in Hematopoiesis and Lymphoid Development between Humans and Mice in JOURNAL OF CLINICAL IMMUNOLOGY
  • 2012-09-02. Lymphoid Priming in Human Bone Marrow Begins Prior to CD10 Expression with Up-Regulation of L-selectin in NATURE IMMUNOLOGY
  • 2010-07-02. Zinc finger nuclease-mediated CCR5 knockout hematopoietic stem cell transplantation controls HIV-1 in vivo in NATURE BIOTECHNOLOGY
  • 2007-06-28. The woodchuck hepatitis virus post-transcriptional regulatory element reduces readthrough transcription from retroviral vectors in GENE THERAPY
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