Molecular Therapy · 2017 · 272 citations · 31 references
Life SciencesGeneticsMolecular GeneticsGene TherapyGenetic EngineeringGenome EditingCrispr-based EngineeringCrispr-cas SystemsGuide Rna DesignOcular Gene DeliveryOcular Gene TherapyTherapeutic Genome EditingAdeno-associated Viral VectorsClustered Regularly Interspaced Short Palindromic Repeats (Crispr)-based MutagenesisCrispr-cas Genome EditingCrispr-cas9 Genome EditingCrispr-cas9 KnockoutGene Editing TherapyGuide Rna-directed EditingLeber Congenital Amaurosis Therapy
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Multiplex Genome Engineering Using CRISPR/Cas Systems
Le Cong, F. Ann Ran, David Cox et al. · Science · 2013 · 15.7K citations
RNA-Guided Human Genome Engineering via Cas9
Prashant Mali, Luhan Yang, Kevin M. Esvelt et al. · Science · 2013 · 9.4K citations · Full text
In vivo genome editing using Staphylococcus aureus Cas9
F. Ann Ran, Le Cong, Winston X. Yan et al. · Nature · 2015 · 2.7K citations · Full text
RNA-programmed genome editing in human cells
Martin Jínek, Alexandra East, Aaron Cheng et al. · eLife · 2013 · 2.2K citations · Full text
Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis
Albert M. Maguire, Francesca Simonelli, Eric A. Pierce et al. · New England Journal of Medicine · 2008 · 2.1K citations · Full text
Medicine, Genetics, Ophthalmology +17