High Titer Lentivector from a Stable Lenti-Producer Efficiently Corrects CD34+ Hematopoietic Stem Cells from Patients with p47phox-Deficient Chronic Granulomatous Disease
نویسندگان
چکیده
منابع مشابه
Self-Inactivating gp91 Lentivector Corrects the Oxidase Defect in NOD/SCID Mouse Repopulating Peripheral Blood Mobilized CD34 Cells from Patients with X-linked Chronic Granulomatous Disease Running head: Lentivector correction of X-linked CGD Scientific section heading: Gene Therapy
HIV-1 derived lentivectors are promising for gene transfer into hematopoietic stem cells, but require pre-clinical in vivo evaluation relevant to specific human diseases. Non-obese diabetic/severe combined immunodeficient (NOD/SCID) mice accept human hematopoietic stem cell grafts providing a unique opportunity for in vivo evaluation of therapies targeting human hematopoietic diseases. We demon...
متن کامل–deficient Chronic Granulomatous Disease
The predominant genetic defect causing p47phox –deficient chronic granulomatous disease (A47 8 CGD) is a GT deletion ( D GT) at the beginning of exon 2. No explanation exists to account for the high incidence of this single mutation causing a rare disease in an unrelated, racially diverse population. In each of 34 consecutive unrelated normal individuals, both the normal and mutant D GT sequenc...
متن کاملGENE THERAPY Third-generation, self-inactivating gp91phox lentivector corrects the oxidase defect in NOD/SCID mouse–repopulating peripheral blood–mobilized CD34 cells from patients with X-linked chronic granulomatous disease
HIV-1–derived lentivectors are promising for gene transfer into hematopoietic stem cells but require preclinical in vivo evaluation relevant to specific human diseases. Nonobese diabetic/severe combined immunodeficient (NOD/SCID) mice accept human hematopoietic stem cell grafts, providing a unique opportunity for in vivo evaluation of therapies targeting human hematopoietic diseases. We demonst...
متن کاملThird-generation, self-inactivating gp91(phox) lentivector corrects the oxidase defect in NOD/SCID mouse-repopulating peripheral blood-mobilized CD34+ cells from patients with X-linked chronic granulomatous disease.
HIV-1-derived lentivectors are promising for gene transfer into hematopoietic stem cells but require preclinical in vivo evaluation relevant to specific human diseases. Nonobese diabetic/severe combined immunodeficient (NOD/SCID) mice accept human hematopoietic stem cell grafts, providing a unique opportunity for in vivo evaluation of therapies targeting human hematopoietic diseases. We demonst...
متن کاملChronic granulomatous disease: overview and hematopoietic stem cell transplantation.
Chronic granulomatous disease (CGD) still causes significant morbidity and mortality. The difficulty in considering high-risk yet curative treatments, such as allogeneic bone marrow transplantation, lies in the unpredictable courses of both CGD and bone marrow transplantation in different patients. Some patients with CGD can have frequent infections, granulomatous or autoimmune disorders necess...
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ژورنال
عنوان ژورنال: Blood
سال: 2015
ISSN: 0006-4971,1528-0020
DOI: 10.1182/blood.v126.23.2036.2036