Reduction of retinal ganglion cell death in mouse models of familial dysautonomia using AAV-mediated gene therapy and splicing modulators.
Schultz A, Cheng SY, Kirchner E, Costello S, Miettinen H, Chaverra M, King C, George L, Zhao X, Narasimhan J, Weetall M, Slaugenhaupt S, Morini E, Punzo C, Lefcort F.
Schultz A, et al. Among authors: cheng sy.
bioRxiv [Preprint]. 2023 May 24:2023.05.22.541535. doi: 10.1101/2023.05.22.541535.
bioRxiv. 2023.
PMID: 37293016
Free PMC article.
Updated.
Preprint.