Bilateral gene therapy in children with autosomal recessive deafness 9: single-arm trial results.
Wang H, Chen Y, Lv J, Cheng X, Cao Q, Wang D, Zhang L, Zhu B, Shen M, Xu C, Xun M, Wang Z, Tang H, Hu S, Cui C, Jiang L, Yin Y, Guo L, Zhou Y, Han L, Gao Z, Zhang J, Yu S, Gao K, Wang J, Chen B, Wang W, Chen ZY, Li H, Shu Y.
Wang H, et al.
Nat Med. 2024 Jul;30(7):1898-1904. doi: 10.1038/s41591-024-03023-5. Epub 2024 Jun 5.
Nat Med. 2024.
PMID: 38839897
Free PMC article.
Clinical Trial.
We recently showed that unilateral AAV1-hOTOF gene therapy with dual adeno-associated virus (AAV) serotype 1 carrying human OTOF transgene is safe and associated with functional improvements in patients with autosomal recessive deafness 9 (DFNB9). The protocol was s …
We recently showed that unilateral AAV1-hOTOF gene therapy with dual adeno-associated virus (AAV) serotype 1 carrying human OTOF transgene i …