Natural variations in AAVHSC16 significantly reduce liver tropism and maintain broad distribution to periphery and CNS.

Autor: Smith LJ; Homology Medicines, Inc., 1 Patriots Park, Bedford, MA 01730, USA., Schulman LA; Homology Medicines, Inc., 1 Patriots Park, Bedford, MA 01730, USA., Smith S; Homology Medicines, Inc., 1 Patriots Park, Bedford, MA 01730, USA., Van Lieshout L; Homology Medicines, Inc., 1 Patriots Park, Bedford, MA 01730, USA., Barnes CM; Homology Medicines, Inc., 1 Patriots Park, Bedford, MA 01730, USA., Behmoiras L; Homology Medicines, Inc., 1 Patriots Park, Bedford, MA 01730, USA., Scarpitti M; Homology Medicines, Inc., 1 Patriots Park, Bedford, MA 01730, USA., Kivaa M; Homology Medicines, Inc., 1 Patriots Park, Bedford, MA 01730, USA., Duong KL; Homology Medicines, Inc., 1 Patriots Park, Bedford, MA 01730, USA., Benard LO; Homology Medicines, Inc., 1 Patriots Park, Bedford, MA 01730, USA., Ellsworth JL; Homology Medicines, Inc., 1 Patriots Park, Bedford, MA 01730, USA., Avila N; Homology Medicines, Inc., 1 Patriots Park, Bedford, MA 01730, USA., Faulkner D; Homology Medicines, Inc., 1 Patriots Park, Bedford, MA 01730, USA., Hayes A; Homology Medicines, Inc., 1 Patriots Park, Bedford, MA 01730, USA., Lotterhand J; Homology Medicines, Inc., 1 Patriots Park, Bedford, MA 01730, USA., Rivas JI; Homology Medicines, Inc., 1 Patriots Park, Bedford, MA 01730, USA., Sengooba AV; Homology Medicines, Inc., 1 Patriots Park, Bedford, MA 01730, USA., Tzianabos A; Homology Medicines, Inc., 1 Patriots Park, Bedford, MA 01730, USA., Seymour AB; Homology Medicines, Inc., 1 Patriots Park, Bedford, MA 01730, USA., Francone OL; Homology Medicines, Inc., 1 Patriots Park, Bedford, MA 01730, USA.

Publikováno v: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2022 Jun 30; Vol. 26, pp. 224-238. Date of Electronic Publication: 2022 Jun 30 (Print Publication: 2022).