Concepedia

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

Divya Sinha, Benjamin Steyer, Pawan K. Shahi, Katherine P. Mueller, Rasa Valiauga, Kimberly L. Edwards, Cole Bacig, Stephanie Steltzer, Sandhya Srinivasan, Amr A. Abdeen,

The American Journal of Human Genetics · 2020 · 58 citations · 35 references

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35

RNA-Guided Human Genome Engineering via Cas9

Prashant Mali, Luhan Yang, Kevin M. Esvelt et al. · Science · 2013 · 9.4K citations · Full text

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Algorithms for Non-negative Matrix Factorization

Daniel D. Lee, H. Sebastian Seung · 2000 · 5.5K citations

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Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

Stephen R. Russell, Jean Bennett, Jennifer Wellman et al. · The Lancet · 2017 · 1.9K citations

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High-throughput profiling of off-target DNA cleavage reveals RNA-programmed Cas9 nuclease specificity

Vikram Pattanayak, Steven Lin, John P. Guilinger et al. · Nature Biotechnology · 2013 · 1.5K citations · Full text

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Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10

Morgan L. Maeder, Michael Stefanidakis, Christopher J. Wilson et al. · Nature Medicine · 2019 · 706 citations

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