Gene therapy studies in a canine model of X-linked severe combined immunodeficiency.
Hum Gene Ther Clin Dev
; 26(1): 50-6, 2015 Mar.
Article
en En
| MEDLINE
| ID: mdl-25603151
Since the occurrence of T cell leukemias in the original human γ-retroviral gene therapy trials for X-linked severe combined immunodeficiency (XSCID), considerable effort has been devoted to developing safer vectors. This review summarizes gene therapy studies performed in a canine model of XSCID to evaluate the efficacy of γ-retroviral, lentiviral, and foamy viral vectors for treating XSCID and a novel method of vector delivery. These studies demonstrate that durable T cell reconstitution and thymopoiesis with no evidence of any serious adverse events and, in contrast to the human XSCID patients, sustained marking in myeloid cells and B cells with reconstitution of normal humoral immune function can be achieved for up to 5 years without any pretreatment conditioning. The presence of sustained levels of gene-marked T cells, B cells, and more importantly myeloid cells for almost 5 years is highly suggestive of transduction of either multipotent hematopoietic stem cells or very primitive committed progenitors.
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Retroviridae
/
Terapia Genética
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Enfermedades por Inmunodeficiencia Combinada Ligada al Cromosoma X
Límite:
Animals
/
Humans
Idioma:
En
Revista:
Hum Gene Ther Clin Dev
Año:
2015
Tipo del documento:
Article
Pais de publicación:
Estados Unidos