Characterization of an MDR1 retroviral bicistronic vector for correction of X-linked severe combined immunodeficiency
نویسندگان
چکیده
منابع مشابه
Retroviral-mediated gene correction for X-linked severe combined immunodeficiency.
X-linked severe combined immunodeficiency (XSCID) is a lethal disease caused by a defect in the gene encoding the common gamma chain (gamma-c) of the receptor for interleukin-2 (IL-2), IL-4, IL-7, IL-9, and IL-15. Allogeneic bone marrow transplantation, the current therapy of choice for this defect, is often complicated by graft-versus-host disease and/or incomplete reconstitution of B-lymphocy...
متن کاملCorrection of canine X-linked severe combined immunodeficiency by in vivo retroviral gene therapy.
X-linked severe combined immunodeficiency (XSCID) is characterized by profound immunodeficiency and early mortality, the only potential cure being hematopoietic stem cell (HSC) transplantation or gene therapy. Current clinical gene therapy protocols targeting HSCs are based upon ex vivo gene transfer, potentially limited by the adequacy of HSC harvest, transduction efficiencies of repopulating ...
متن کاملGENE THERAPY Correction of canine X-linked severe combined immunodeficiency by in vivo retroviral gene therapy
X-linked severe combined immunodeficiency (XSCID) is characterized by profound immunodeficiency and early mortality, the only potential cure being hematopoietic stem cell (HSC) transplantation or gene therapy. Current clinical gene therapy protocols targeting HSCs are based upon ex vivo gene transfer, potentially limited by the adequacy of HSC harvest, transduction efficiencies of repopulating ...
متن کاملEstablishment of modified retroviral vector targeting X-linked severe combined immunodeficiency.
Gene therapy targeting hematopoietic stem cells has been proposed as a potential therapy for numerous genetic disorders affecting hematopoiesis. Moloney murine leukemia retroviral vectors are now widely used for clinical gene transfer into hematopoietic progenitors and progeny. However, maintaining expression of therapeutic genes inserted via moloney murine leukemia virus (MoMLV)-based vectors ...
متن کاملEfficacy of gene therapy for X-linked severe combined immunodeficiency.
BACKGROUND The outcomes of gene therapy to correct congenital immunodeficiencies are unknown. We reviewed long-term outcomes after gene therapy in nine patients with X-linked severe combined immunodeficiency (SCID-X1), which is characterized by the absence of the cytokine receptor common gamma chain. METHODS The nine patients, who lacked an HLA-identical donor, underwent ex vivo retrovirus-me...
متن کاملذخیره در منابع من
با ذخیره ی این منبع در منابع من، دسترسی به آن را برای استفاده های بعدی آسان تر کنید
ژورنال
عنوان ژورنال: Gene Therapy
سال: 1998
ISSN: 0969-7128,1476-5462
DOI: 10.1038/sj.gt.3300651