Molecular BiologyProceedings of the National Academy of SciencesPeer-reviewed
Characterisation of RNA editing and gene therapy with a compact CRISPR-Cas13 in the retina
Satheesh Kumar, Yi-Wen Hsiao, Vickie H. Y. Wong…·Guei‐Sheung Liu
This study characterizes the transcriptomic effects of Cas13bt3 in retinal cells and demonstrates its efficacy in silencing VEGFA mRNA in human retinal organoids and mouse models.