Marcella GARITA-HERNANDEZ
Head of iPS Core Harvard Medical School
Seminars
Wednesday 9th September 2026
Accelerating Ocular Diseases Therapeutics: Gene Editing in iPSC-Derived Retinal Organoids
4:10 pm
- Field overview of patient-derived iPSCs and retinal organoids, which provide a disease-relevant, human preclinical platform enabling functional validation of gene editing and cell therapies directly in photoreceptors, overcoming limitations of animal models
- Functional recovery validates therapeutic potential: restoration of key phototransduction components (e.g., PDE6, RHO, and others) in organoids confirms that genetic correction translates into biologically meaningful rescue, supporting singledose, gene and mutation-targeted therapies
- This approach helps to evaluate the efficacy, delivery strategies, and safety of genome-editing therapies across diverse monogenic diseases, supporting both regulatory decision-making and therapeutic development pipelines
