SEP 9, 2026 · JOURNAL ARTICLE
CRISPR and Gene Augmentation Rescue Trabecular Meshwork Dysfunction in iPSC Models of Lowe Syndrome
Advanced Science
In vitro proof-of-concept in iPSC-derived cell models demonstrating feasibility of two gene therapy approaches, without in vivo efficacy, clinical outcomes, or animal validation.
Study details
DesignIn vitro mechanistic proof-of-concept study
InterventionAAV2-OCRL gene augmentation therapy and pat…
ComparatorPatient-derived iPSC trabecular meshwork mo…