Intra-CNS AAV9- ⁇ i>GBA1 ⁇ /i> teslimi, güvenlik ve transgen ifadesinde yönetim farklılıklarının tür ve rotasını verir.

Intra-CNS AAV9-<i>GBA1</i> delivery yields species and route of administration differences in safety and transgene expression.

Amaral AC, Grubor B, Gianni D, Koetzner L, Abraham N, Bourque S, Brown D, Chen Y, Chicoine KE, Clarner P, De Giovanni PJ, Hamann S, Kirkland M, Mendes OR, Michael M, Nadella MVP, Nambiar K, Sebalusky J, Zeng W, Xu S, Trapa P, Plowey ED, Tien E, Fikes J, Walsh DM, Hirst WD, Suh J, Glajch KE.

Mol Ther Adv · 2026-06-11

DOI: 10.1016/j.omta.2026.201779PMID: 42422766