Molecular BiologybioRxivPreprint
Using RNA-targeting CRISPR-Cas13 and engineered U1 systems to reduce ABCA4 splice variants in Stargardt disease
Roxanne Hsiang-Chi Liou, Daniel Urrutia-Cabrera, Ida Maria Westin…·Raymond C.B. Wong
This study evaluates RNA-targeting CRISPR-Cas13 and engineered U1 systems to reduce mis-spliced ABCA4 variants in Stargardt disease, showing significant reductions in disease-causing splice variants.
DOI 10.1101/2024.03.08.584155