Spinal subpial delivery of AAV9 enables widespread gene silencing and blocks motoneuron degeneration in ALS.
Nat Med. 2020 Jan;26(1):118-130. doi: 10.1038/s41591-019-0674-1. Epub 2019 Dec 23.
Nat Med. 2020.
PMID: 31873312
Free PMC article.