Human iPSC Modeling Reveals Mutation-Specific Responses to Gene Therapy in a Genotypically Diverse Dominant Maculopathy

Gamm, MD, PhD, D. Human IPSC Modeling Reveals Mutation-Specific Responses to Gene Therapy in a Genotypically Diverse Dominant Maculopathy.
Sinha, D., Steyer, B., Shahi, P. K., Mueller, K. P., Valiauga, R., Edwards, K. L., Bacig, C., Steltzer, S. S., Srinivasan, S., Abdeen, A., Cory, E., Periyasamy, V., Siahpirani, A. F., Stone, E. M., Tucker, B. A., Roy, S., Pattnaik, B. R., Saha, K., & Gamm, D. M. (2020). Human iPSC Modeling Reveals Mutation-Specific Responses to Gene Therapy in a Genotypically Diverse Dominant Maculopathy. American journal of human genetics, 107(2), 278–292. https://doi.org/10.1016/j.ajhg.2020.06.011