Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency

X-linked severe combined immunodeficiency (SCID-X1) is caused by mutations in the interleukin-2 receptor γ chain gene (IL2RG), and it is characterized by profound defects in T, B, and natural killer (NK) cell functions. Transplantation of hematopoietic stem/progenitor cells (HSPCs) genetically corre...

Descripción completa

Detalles Bibliográficos
Autores principales: Poletti, Valentina, Charrier, Sabine, Corre, Guillaume, Gjata, Bernard, Vignaud, Alban, Zhang, Fang, Rothe, Michael, Schambach, Axel, Gaspar, H. Bobby, Thrasher, Adrian J., Mavilio, Fulvio
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5918176/
https://www.ncbi.nlm.nih.gov/pubmed/29707600
http://dx.doi.org/10.1016/j.omtm.2018.03.002

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5918176/
https://www.ncbi.nlm.nih.gov/pubmed/29707600
http://dx.doi.org/10.1016/j.omtm.2018.03.002

Ejemplares similares