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: kirchner e.
Sci Rep. 2023 Oct 30;13(1):18600. doi: 10.1038/s41598-023-45376-w.
Sci Rep. 2023.
PMID: 37903840
Free PMC article.