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

https://doi.org/10.1016/j.ajhg.2020.06.011

Sinha, Divya ; Steyer, Benjamin ; Shahi, Pawan K. ; Mueller, Katherine P. ; Valiauga, Rasa ; Edwards, Kimberly L. ; Bacig, Cole ; Steltzer, Stephanie S. ; Srinivasan, Sandhya ; Abdeen, Amr ; et al ( August 2020 , The American Journal of Human Genetics)
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Scarless Genome Editing of Human Pluripotent Stem Cells via Transient Puromycin Selection

https://doi.org/10.1016/j.stemcr.2017.12.004

Steyer, Benjamin ; Bu, Qian ; Cory, Evan ; Jiang, Keer ; Duong, Stella ; Sinha, Divya ; Steltzer, Stephanie ; Gamm, David ; Chang, Qiang ; Saha, Krishanu ( February 2018 , Stem Cell Reports)
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