Precise in vivo genome editing via single homology arm donor mediated intron-targeting gene integration for genetic disease correction
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We are grateful to M.Schwarz and P.Schwarz for administrative help;D.O”Keefe and S.Tsuji for help with manuscript preparation;M.Kay and Z.Y.Chen for sharing experimental materials;J.Naughton and J.Marlett for AAV production;K.Peterson and Y.Gu for helping the measurement of heart rate;U.Manor and K.Diffenderfer for imaging;K.Mclntyre for mouse histology processing and J.Li for helping the molecular work;K.Sumiyama for data analysis.M.Y.was partially supported by 2016 Salk Women & Science Special Award.K.S.was supported by JSPS KAKENHI15K21762 and 18H04036;Takeda Science Foundation,The Uehara Memorial Foundation,National Institutes of Natural SciencesBS291007;The Sumitomo Foundation170220;The Naito Foundation,The Kurata Grants1350;Mochida Memorial Foundation,and The Inamori Foundation.This research was supported by Guangdong Provincial Key Laboratory of Genome Read and Write2017B030301011;Guangdong Provincial Academician Workstation of BGI Synthetic Genomics2017B090904014;Shenzhen Peacock Plan No.KQTD20150330171505310.J.C.I.B.was supported by The Leona M.and Harry B.Helmsley Charitable Trust2012-PG-MED002;the G.Harold and Leila Y.Mathers Charitable Foundation,NIHR01HL123755 and 5 DP1 DK113616;The Progeria Research Foundation,The Glenn Foundation,KAUST,The Moxie Foundation,Fundación Dr.Pedro Guillen,AFE and Universidad Católica San Antonio de Murcia
2019-12-11（万方平台首次上网日期，不代表论文的发表时间）
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