Human iPSC Modeling Reveals Mutation-Specific Responses to Gene Therapy in a Genotypically Diverse Dominant Maculopathy
人类 iPSC 建模揭示了基因型多样化显性黄斑病变对基因治疗的突变特异性反应
期刊:American Journal of Human Genetics
影响因子:8.1
doi:10.1016/j.ajhg.2020.06.011
Divya Sinha, Benjamin Steyer, Pawan K Shahi, Katherine P Mueller, Rasa Valiauga, Kimberly L Edwards, Cole Bacig, Stephanie S Steltzer, Sandhya Srinivasan, Amr Abdeen, Evan Cory, Viswesh Periyasamy, Alireza Fotuhi Siahpirani, Edwin M Stone, Budd A Tucker, Sushmita Roy, Bikash R Pattnaik, Krishanu Sah