CRISPR and Gene Augmentation Rescue Trabecular Meshwork Dysfunction in iPSC Models of Lowe Syndrome
The results showed that AAV2-OCRL demonstrated the highest transduction efficiency in patient iPSC-derived trabecular meshwork models (iHTM) among the three AAV-OCRL vectors evaluated, establishing it as a promising delivery vector.
Si-Yu Chen, Zhi-Quan Liu, Wen-Min Wang et al.
· Advancement of science · 0 citations