Sustained Correction of a Murine Model of Phenylketonuria following a Single Intravenous Administration of AAVHSC15-PAH.

Autor: Ahmed SS; Research and Development, Homology Medicines, 1 Patriots Park, Bedford, MA 01730, USA., Rubin H; Research and Development, Homology Medicines, 1 Patriots Park, Bedford, MA 01730, USA., Wang M; Research and Development, Homology Medicines, 1 Patriots Park, Bedford, MA 01730, USA., Faulkner D; In Vivo Group, Homology Medicines, 1 Patriots Park, Bedford, MA 01730, USA., Sengooba A; In Vivo Group, Homology Medicines, 1 Patriots Park, Bedford, MA 01730, USA., Dollive SN; Research and Development, Homology Medicines, 1 Patriots Park, Bedford, MA 01730, USA., Avila N; In Vivo Group, Homology Medicines, 1 Patriots Park, Bedford, MA 01730, USA., Ellsworth JL; Research and Development, Homology Medicines, 1 Patriots Park, Bedford, MA 01730, USA., Lamppu D; Program Management Group, Homology Medicines, 1 Patriots Park, Bedford, MA 01730, USA., Lobikin M; Process Development, Homology Medicines, 1 Patriots Park, Bedford, MA 01730, USA., Lotterhand J; In Vivo Group, Homology Medicines, 1 Patriots Park, Bedford, MA 01730, USA., Adamson-Small L; Process Development, Homology Medicines, 1 Patriots Park, Bedford, MA 01730, USA., Wright T; Toxicology Group, Homology Medicines, 1 Patriots Park, Bedford, MA 01730, USA., Seymour A; Research and Development, Homology Medicines, 1 Patriots Park, Bedford, MA 01730, USA., Francone OL; Research and Development, Homology Medicines, 1 Patriots Park, Bedford, MA 01730, USA.

Publikováno v: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2020 Mar 13; Vol. 17, pp. 568-580. Date of Electronic Publication: 2020 Mar 13 (Print Publication: 2020).