Human iPSC modeling reveals mutation-specific responses to gene therapy in Best disease
Divya Sinha, Benjamin Steyer, Pawan Kumar Shahi, Katherine P. Mueller, Rasa Valiauga, Kimberly L. Edwards, Cole Bacig, Stephanie S. Steltzer, Sandhya Srinivasan, Amr A. Abdeen, Evan Cory, Viswesh Periyasamy, Alireza Fotuhi Siahpirani, Edwin M. Stone, Budd A. Tucker, , David M. Gamm
This study uses iPSC-RPE models to evaluate gene augmentation and editing for Best disease, finding that some mutations respond to gene therapy while others require genome editing.
bioRxiv · Jan 31, 2020 · PreprintGenetics