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Precise in vivo genome editing via single homology arm donor mediated intron-targeting gene integration for genetic disease correction

查看全文 作　　者：Keiichiro [1,2,3]Suzuki;Mako [1]Yamamoto;Reyna Hernandez-[1]Benitez;Zhe [4]Li;Christopher [4]Wei;Rupa Devi [1,5]Soligalla;Emi [1,3]Aizawa;Fumiyuki [1]Hatanaka;Masakazu [1,5]Kurita;Pradeep [1]Reddy;Alejandro [1]Ocampo;Tomoaki [1]Hishida;Masahiro [1,5]Sakurai;Amy [1]NNemeth;Estrella Nunez [5]Delicado;Josep [6]MCampistol;Pierre [7]Magistretti;Pedro [8]Guillen;Concepcion Rodriguez [1]Esteban;Jianhui [9,10,11,12]Gong;Yilin [9,10,11]Yuan;Ying [9,10,11]Gu;Guang-Hui [13]Liu;Carlos Lopez-[14]Otin;Jun [5,15,16]Wu;Kun [4]Zhang;Juan Carlos Izpisua [1]Belmonte 高影响力作者 机构地区：[1]Gene Expression Laboratory,Salk Institute for Biological Studies,La Jolla,CA 92037,USA;[2]Institute for Advanced Co-Creation Studies,Osaka University,Osaka 560-8531,Japan;[3]Graduate School of Engineering Science,Osaka University,Osaka 560-8531,Japan;[4]Bioengineering,University of California,San Diego,9500 Gilman Drive,MC0412,La Jolla,CA92093-0412,USA;[5]Universidad Catolica,San Antonio de Murcia,Campus de los Jeronimos,135,Guadalupe 30107,Spain;[6]Hospital Clinic of Barcelona,Carrer Villarroel,170,08036 Barcelona,Spain;[7]King Abdullah University of Science and Technology(KAUST),Thuwal 23955-6900,Saudi Arabia;[8]Fundacion Dr.Pedro Guillen,Clinica CEMTRO,Avenida Ventisquero de la Condesa,4228035 Madrid,Spain;[9]BGI-Shenzhen,Shenzhen 518083,China;[10]Guangdong Provincial Key Laboratory of Genome Read and Write,Shenzhen 518120,China;[11]Guangdong Provincial Academician Workstation of BGI Synthetic Genomics,BGI Shenzhen,Guangdong,China;[12]Shenzhen Engineering Laboratory for Innovative Molecular Diagnostics,Shenzhen 518120,China;[13]National Laboratory of Biomacromolecules,CAS Center for Excellence in Biomacromolecules,Institute of Biophysics,Chinese Academy of Sciences,Beijing 100101,China;[14]Departamento de Bioqufmica y Biologia Molecular,Facultad de Medicina,Instituto Universitario de Oncologfa(IUOPA),Universidad de Oviedo,Oviedo,Spain;[15]Department of Molecular Biology,University of Texas Southwestern Medical Center,Dallas,TX 75390,USA;[16]Hamon Center for Regenerative Science and Medicine,University of Texas Southwestern Medical Center,Dallas,TX 75390,USA高影响力机构 出　　处：《Cell Research》索引2019年第29卷第10期,共16页高影响力期刊 基　　金：JSPS KAKENHI(15K21762 and 18H04036);Takeda Science Foundation;The Uehara Memorial Foundation;National Institutes of Natural Sciences(BS291007);The Sumitomo Foundation(170220);The Naito Foundation;The Kurata Grants(1350);Mochida Memorial Foundation;The Inamori Foundation.This research was supported by Guangdong Provincial Key Laboratory of Genome Read and Write(No.2017B030301011);Guangdong Provincial Academician Workstation of BGI Synthetic Genomics(No.2017B090904014);Shenzhen Peacock Plan(No.KQTD20150330171505310).J.C.I.B.was supported by The Leona M.and Harry B.Helmsley Charitable Trust(2012-PG-MED002);the G.Harold and Leila Y.Mathers Charitable Foundation;NIH(R01HL123755 and 5 DP1 DK113616);The Progeria Research Foundation;The Glenn Foundation,KAUST,The Moxie Foundation;Fundacion Dr.Pedro Guillen;AFE and Universidad Catolica San Antonio de Murcia(UCAM). 摘　　要：In vivo genome editing represents a powerful strategy for both understanding basic biology and treating inherited diseases.However,it remains a challenge to develop universal and efficient in vivo genome-editing tools for tissues that comprise diverse cell types in either a dividing or non-dividing state.Here,we describe a versatile in vivo gene knock-in methodology that enables the targeting of a broad range of mutations and cell types through the insertion of a minigene at an intron of the target gene locus using an intracellularly linearized single homology arm donor.As a proof-of-concept,we focused on a mouse model of prematureaging caused by a dominant point mutation,which is difficult to repair using existing in vivo genome-editing tools.Systemic treatment using our new method ameliorated aging-associated phenotypes and extended animal lifespan,thus highlighting the potential of this methodology for a broad range of in vivo genome-editing applications. 关 键 词：VIVO DONOR dividing
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