Biblio
CRISPR-Cas9 correction of a nonsense mutation in rescues lebercilin expression and localization in human retinal organoids. Mol Ther Methods Clin Dev. 2023;29:522-531.
Detailed Phenotyping and Therapeutic Strategies for Intronic ABCA4 Variants in Stargardt Disease. Mol Ther Nucleic Acids. 2020;21:412-427.
Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human c.2991+1655A>G LCA10 Models. Mol Ther Nucleic Acids. 2018;12:730-740.