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Highly Efficient Correction of the Sickle Cell Disease Mutation in Patient HSC Using an RNA Gene Writing System, an RNA-Based, Nuclease-Free Approach to Genome Editing

Giulia Schiroli, Yaser Heshmati,Aamir Mir, Yu Cao, Shallu Kutlehria, Jicheng Duan, Lorenzo Tozzi, Rui Zhang, Felipe Gazos Lopez, William E. Salomon,James B. Rottman,Jane Wang,Anne Bothmer,Jean-Antoine Ribeil,John P. Manis,Cecilia Cotta-Ramusino,Michael C. Holmes

MOLECULAR THERAPY(2023)

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