Immune transgene-dependent myocarditis in macaques after systemic administration of adeno-associated virus expressing human acid alpha-glucosidase.
Hordeaux J, Ramezani A, Tuske S, Mehta N, Song C, Lynch A, Lupino K, Chichester JA, Buza EL, Dyer C, Yu H, Bell P, Weimer JM, Do H, Wilson JM.
Hordeaux J, et al.
Front Immunol. 2023 Mar 22;14:1094279. doi: 10.3389/fimmu.2023.1094279. eCollection 2023.
Front Immunol. 2023.
PMID: 37033976
Free PMC article.
We developed a gene therapy candidate for Pompe disease consisting of AAVhu68, a clade F AAV closely related to AAV9, that expresses an engineered human acid-alpha glucosidase (hGAA) tagged with an insulin-like growth factor 2 variant (vIGF2) peptide for enhanced cell upta …
We developed a gene therapy candidate for Pompe disease consisting of AAVhu68, a clade F AAV closely related to AAV9, that expresses …