Journal article
The NIH Somatic Cell Genome Editing program
Nature (London), Vol.592(7853), pp.195-204
04/2021
DOI: 10.1038/s41586-021-03191-1
PMCID: PMC8026397
PMID: 33828315
Abstract
The move from reading to writing the human genome offers new opportunities to improve human health. The United States National Institutes of Health (NIH) Somatic Cell Genome Editing (SCGE) Consortium aims to accelerate the development of safer and more-effective methods to edit the genomes of disease-relevant somatic cells in patients, even in tissues that are difficult to reach. Here we discuss the consortium's plans to develop and benchmark approaches to induce and measure genome modifications, and to define downstream functional consequences of genome editing within human cells. Central to this effort is a rigorous and innovative approach that requires validation of the technology through third-party testing in small and large animals. New genome editors, delivery technologies and methods for tracking edited cells in vivo, as well as newly developed animal models and human biological systems, will be assembled-along with validated datasets-into an SCGE Toolkit, which will be disseminated widely to the biomedical research community. We visualize this toolkit-and the knowledge generated by its applications-as a means to accelerate the clinical development of new therapies for a wide range of conditions.
Details
- Title: Subtitle
- The NIH Somatic Cell Genome Editing program
- Creators
- Krishanu Saha - University of Wisconsin–MadisonErik J Sontheimer - University of Massachusetts Chan Medical SchoolP J Brooks - National Institutes of HealthMelinda R Dwinell - Medical College of WisconsinCharles A Gersbach - Duke UniversityDavid R Liu - Broad InstituteStephen A Murray - Jackson LaboratoryShengdar Q Tsai - St. Jude Children's Research HospitalRoss C Wilson - University of California, BerkeleyDaniel G Anderson - Massachusetts Institute of TechnologyAravind Asokan - Duke UniversityJillian F Banfield - University of California, BerkeleyKrystof S Bankiewicz - The Ohio State UniversityGang Bao - Rice UniversityJeff W M Bulte - Johns Hopkins MedicineNenad Bursac - Duke UniversityJarryd M Campbell - Recombinetics (United States)Daniel F Carlson - Recombinetics (United States)Elliot L Chaikof - Beth Israel Deaconess Medical CenterZheng-Yi Chen - Harvard UniversityR Holland Cheng - University of California, DavisKarl J Clark - Mayo ClinicDavid T Curiel - Washington University in St. LouisJames E Dahlman - Georgia Institute of TechnologyBenjamin E Deverman - Broad InstituteMary E Dickinson - Baylor College of MedicineJennifer A Doudna - Gladstone InstitutesStephen C Ekker - Mayo ClinicMarina E Emborg - University of Wisconsin–MadisonGuoping Feng - McGovern Institute for Brain ResearchBenjamin S Freedman - University of WashingtonDavid M Gamm - University of Wisconsin–MadisonGuangping Gao - University of Massachusetts Chan Medical SchoolIonita C Ghiran - Beth Israel Deaconess Medical CenterPeter M Glazer - Yale UniversityShaoqin Gong - University of Wisconsin–MadisonJason D Heaney - Baylor College of MedicineJon D Hennebold - Oregon National Primate Research CenterJohn T Hinson - University of ConnecticutAnastasia Khvorova - University of Massachusetts Chan Medical SchoolSamira Kiani - University of PittsburghWilliam R Lagor - Baylor College of MedicineKit S Lam - University of California, DavisKam W Leong - Columbia UniversityJon E Levine - University of Wisconsin–MadisonJennifer A Lewis - Harvard UniversityCathleen M Lutz - Jackson LaboratoryDanith H Ly - Carnegie Mellon UniversitySamantha Maragh - National Institute of Standards and TechnologyPaul B McCray Jr - Department of Pediatrics, University of Iowa, Iowa City, IA, USATodd C McDevitt - University of California, San FranciscoOleg Mirochnitchenko - National Institutes of HealthRyuji Morizane - Harvard UniversityNiren Murthy - University of California, BerkeleyRandall S Prather - University of MissouriJohn A Ronald - Western UniversitySubhojit Roy - University of California San DiegoSushmita Roy - University of Wisconsin–MadisonVenkata Sabbisetti - Brigham and Women's HospitalW Mark Saltzman - Yale UniversityPhilip J Santangelo - Georgia Institute of TechnologyDavid J Segal - University of California, DavisMary Shimoyama - Medical College of WisconsinMelissa C Skala - Morgridge Institute for ResearchAlice F Tarantal - University of California, DavisJohn C Tilton - Case Western Reserve UniversityGeorge A Truskey - Duke UniversityMoriel Vandsburger - University of California, BerkeleyJonathan K Watts - University of Massachusetts Chan Medical SchoolKevin D Wells - University of MissouriScot A Wolfe - University of Massachusetts Chan Medical SchoolQiaobing Xu - Tufts UniversityWen Xue - University of Massachusetts Chan Medical SchoolGuohua Yi - The University of Texas at AustinJiangbing Zhou - Yale UniversitySCGE Consortium
- Resource Type
- Journal article
- Publication Details
- Nature (London), Vol.592(7853), pp.195-204
- DOI
- 10.1038/s41586-021-03191-1
- PMID
- 33828315
- PMCID
- PMC8026397
- NLM abbreviation
- Nature
- ISSN
- 0028-0836
- eISSN
- 1476-4687
- Grant note
- UH3 EB028910 / NIBIB NIH HHS UH2 EB028910 / NIBIB NIH HHS U42 OD026645 / NIH HHS U01 HL145792 / NHLBI NIH HHS UG3 AI150551 / NIAID NIH HHS U42 OD026635 / NIH HHS R35 GM119644 / NIGMS NIH HHS UH3 NS111688 / NINDS NIH HHS UH3 HL147352 / NHLBI NIH HHS U42 OD027094 / NIH HHS UH2 EB028907 / NIBIB NIH HHS UG3 HL151545 / NHLBI NIH HHS U01 DK127587 / NIDDK NIH HHS U01 ES032673 / NIEHS NIH HHS DP2 DK133821 / NIDDK NIH HHS P51 OD011107 / NIH HHS UC2 DK126023 / NIDDK NIH HHS S10 OD028713 / NIH HHS R56 DK122380 / NIDDK NIH HHS UG3 NS111688 / NINDS NIH HHS P51 OD011106 / NIH HHS P01 HL152960 / NHLBI NIH HHS U24 HG010423 / NHGRI NIH HHS UG3 HL147352 / NHLBI NIH HHS U01 EB029371 / NIBIB NIH HHS U01 AI145965 / NIAID NIH HHS U01 EB028899 / NIBIB NIH HHS U01 EY032333 / NEI NIH HHS
- Language
- English
- Date published
- 04/2021
- Academic Unit
- Microbiology and Immunology; Pulmonary Medicine; Stead Family Department of Pediatrics; Internal Medicine
- Record Identifier
- 9984297323202771
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