Ontology highlight
ABSTRACT:
SUBMITTER: Gernoux G
PROVIDER: S-EPMC7054721 | biostudies-literature | 2020 Mar
REPOSITORIES: biostudies-literature
Gernoux Gwladys G Gruntman Alisha M AM Blackwood Meghan M Zieger Marina M Flotte Terence R TR Mueller Christian C
Molecular therapy : the journal of the American Society of Gene Therapy 20200113 3
With the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) approvals for Zolgensma, Luxturna, and Glybera, recombinant adeno-associated viruses (rAAVs) are considered efficient tools for gene transfer. However, studies in animals and humans demonstrate that intramuscular (IM) AAV delivery can trigger immune responses to AAV capsids and/or transgenes. IM delivery of rAAV1 in humans has also been described to induce tolerance to rAAV characterized by the presence of capsid- ...[more]