Ontology highlight
ABSTRACT:
SUBMITTER: George LA
PROVIDER: S-EPMC8877054 | biostudies-literature | 2021 Dec
REPOSITORIES: biostudies-literature
Hematology. American Society of Hematology. Education Program 20211201 1
After 3 decades of clinical trials, repeated proof-of-concept success has now been demonstrated in hemophilia A and B gene therapy. Current clinical hemophilia gene therapy efforts are largely focused on the use of systemically administered recombinant adeno-associated viral (rAAV) vectors for F8 or F9 gene addition. With multiple ongoing trials, including licensing studies in hemophilia A and B, many are cautiously optimistic that the first AAV vectors will obtain regulatory approval within app ...[more]