Ontology highlight
ABSTRACT:
SUBMITTER: Humbert O
PROVIDER: S-EPMC5942001 | biostudies-literature | 2018 May
REPOSITORIES: biostudies-literature
Humbert Olivier O Chan Frieda F Rajawat Yogendra S YS Torgerson Troy R TR Burtner Christopher R CR Hubbard Nicholas W NW Humphrys Daniel D Norgaard Zachary K ZK O'Donnell Patricia P Adair Jennifer E JE Trobridge Grant D GD Scharenberg Andrew M AM Felsburg Peter J PJ Rawlings David J DJ Kiem Hans-Peter HP
Blood advances 20180501 9
Hematopoietic stem-cell gene therapy is a promising treatment of X-linked severe combined immunodeficiency disease (SCID-X1), but currently, it requires recipient conditioning, extensive cell manipulation, and sophisticated facilities. With these limitations in mind, we explored a simpler therapeutic approach to SCID-X1 treatment by direct IV administration of foamy virus (FV) vectors in the canine model. FV vectors were used because they have a favorable integration site profile and are resista ...[more]