Literature DB >> 34710352

Imagine CRISPR cures.

Fyodor D Urnov1.   

Abstract

Entities:  

Mesh:

Year:  2021        PMID: 34710352      PMCID: PMC8571165          DOI: 10.1016/j.ymthe.2021.10.019

Source DB:  PubMed          Journal:  Mol Ther        ISSN: 1525-0016            Impact factor:   11.454


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  15 in total

1.  In vivo genome editing of the albumin locus as a platform for protein replacement therapy.

Authors:  Rajiv Sharma; Xavier M Anguela; Yannick Doyon; Thomas Wechsler; Russell C DeKelver; Scott Sproul; David E Paschon; Jeffrey C Miller; Robert J Davidson; David Shivak; Shangzhen Zhou; Julianne Rieders; Philip D Gregory; Michael C Holmes; Edward J Rebar; Katherine A High
Journal:  Blood       Date:  2015-08-21       Impact factor: 22.113

2.  CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia.

Authors:  Haydar Frangoul; David Altshuler; M Domenica Cappellini; Yi-Shan Chen; Jennifer Domm; Brenda K Eustace; Juergen Foell; Josu de la Fuente; Stephan Grupp; Rupert Handgretinger; Tony W Ho; Antonis Kattamis; Andrew Kernytsky; Julie Lekstrom-Himes; Amanda M Li; Franco Locatelli; Markus Y Mapara; Mariane de Montalembert; Damiano Rondelli; Akshay Sharma; Sujit Sheth; Sandeep Soni; Martin H Steinberg; Donna Wall; Angela Yen; Selim Corbacioglu
Journal:  N Engl J Med       Date:  2020-12-05       Impact factor: 91.245

Review 3.  Gene therapy comes of age.

Authors:  Cynthia E Dunbar; Katherine A High; J Keith Joung; Donald B Kohn; Keiya Ozawa; Michel Sadelain
Journal:  Science       Date:  2018-01-12       Impact factor: 47.728

4.  Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease.

Authors:  Jinkuk Kim; Chunguang Hu; Christelle Moufawad El Achkar; Lauren E Black; Julie Douville; Austin Larson; Mary K Pendergast; Sara F Goldkind; Eunjung A Lee; Ashley Kuniholm; Aubrie Soucy; Jai Vaze; Nandkishore R Belur; Kristina Fredriksen; Iva Stojkovska; Alla Tsytsykova; Myriam Armant; Renata L DiDonato; Jaejoon Choi; Laura Cornelissen; Luis M Pereira; Erika F Augustine; Casie A Genetti; Kira Dies; Brenda Barton; Lucinda Williams; Benjamin D Goodlett; Bobbie L Riley; Amy Pasternak; Emily R Berry; Kelly A Pflock; Stephen Chu; Chantal Reed; Kimberly Tyndall; Pankaj B Agrawal; Alan H Beggs; P Ellen Grant; David K Urion; Richard O Snyder; Susan E Waisbren; Annapurna Poduri; Peter J Park; Al Patterson; Alessandra Biffi; Joseph R Mazzulli; Olaf Bodamer; Charles B Berde; Timothy W Yu
Journal:  N Engl J Med       Date:  2019-10-09       Impact factor: 91.245

5.  A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity.

Authors:  Martin Jinek; Krzysztof Chylinski; Ines Fonfara; Michael Hauer; Jennifer A Doudna; Emmanuelle Charpentier
Journal:  Science       Date:  2012-06-28       Impact factor: 47.728

6.  Unconstrained genome targeting with near-PAMless engineered CRISPR-Cas9 variants.

Authors:  Russell T Walton; Kathleen A Christie; Madelynn N Whittaker; Benjamin P Kleinstiver
Journal:  Science       Date:  2020-03-26       Impact factor: 47.728

7.  Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency.

Authors:  Donald B Kohn; Claire Booth; Kit L Shaw; Jinhua Xu-Bayford; Elizabeth Garabedian; Valentina Trevisan; Denise A Carbonaro-Sarracino; Kajal Soni; Dayna Terrazas; Katie Snell; Alan Ikeda; Diego Leon-Rico; Theodore B Moore; Karen F Buckland; Ami J Shah; Kimberly C Gilmour; Satiro De Oliveira; Christine Rivat; Gay M Crooks; Natalia Izotova; John Tse; Stuart Adams; Sally Shupien; Hilory Ricketts; Alejandra Davila; Chilenwa Uzowuru; Amalia Icreverzi; Provaboti Barman; Beatriz Campo Fernandez; Roger P Hollis; Maritess Coronel; Allen Yu; Krista M Chun; Christian E Casas; Ruixue Zhang; Serena Arduini; Frances Lynn; Mahesh Kudari; Andrea Spezzi; Marco Zahn; Rene Heimke; Ivan Labik; Roberta Parrott; Rebecca H Buckley; Lilith Reeves; Kenneth Cornetta; Robert Sokolic; Michael Hershfield; Manfred Schmidt; Fabio Candotti; Harry L Malech; Adrian J Thrasher; H Bobby Gaspar
Journal:  N Engl J Med       Date:  2021-05-11       Impact factor: 91.245

Review 8.  The NIH Somatic Cell Genome Editing program.

Authors:  Krishanu Saha; Erik J Sontheimer; P J Brooks; Melinda R Dwinell; Charles A Gersbach; David R Liu; Stephen A Murray; Shengdar Q Tsai; Ross C Wilson; Daniel G Anderson; Aravind Asokan; Jillian F Banfield; Krystof S Bankiewicz; Gang Bao; Jeff W M Bulte; Nenad Bursac; Jarryd M Campbell; Daniel F Carlson; Elliot L Chaikof; Zheng-Yi Chen; R Holland Cheng; Karl J Clark; David T Curiel; James E Dahlman; Benjamin E Deverman; Mary E Dickinson; Jennifer A Doudna; Stephen C Ekker; Marina E Emborg; Guoping Feng; Benjamin S Freedman; David M Gamm; Guangping Gao; Ionita C Ghiran; Peter M Glazer; Shaoqin Gong; Jason D Heaney; Jon D Hennebold; John T Hinson; Anastasia Khvorova; Samira Kiani; William R Lagor; Kit S Lam; Kam W Leong; Jon E Levine; Jennifer A Lewis; Cathleen M Lutz; Danith H Ly; Samantha Maragh; Paul B McCray; Todd C McDevitt; Oleg Mirochnitchenko; Ryuji Morizane; Niren Murthy; Randall S Prather; John A Ronald; Subhojit Roy; Sushmita Roy; Venkata Sabbisetti; W Mark Saltzman; Philip J Santangelo; David J Segal; Mary Shimoyama; Melissa C Skala; Alice F Tarantal; John C Tilton; George A Truskey; Moriel Vandsburger; Jonathan K Watts; Kevin D Wells; Scot A Wolfe; Qiaobing Xu; Wen Xue; Guohua Yi; Jiangbing Zhou
Journal:  Nature       Date:  2021-04-07       Impact factor: 69.504

9.  CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis.

Authors:  Julian D Gillmore; Ed Gane; Jorg Taubel; Justin Kao; Marianna Fontana; Michael L Maitland; Jessica Seitzer; Daniel O'Connell; Kathryn R Walsh; Kristy Wood; Jonathan Phillips; Yuanxin Xu; Adam Amaral; Adam P Boyd; Jeffrey E Cehelsky; Mark D McKee; Andrew Schiermeier; Olivier Harari; Andrew Murphy; Christos A Kyratsous; Brian Zambrowicz; Randy Soltys; David E Gutstein; John Leonard; Laura Sepp-Lorenzino; David Lebwohl
Journal:  N Engl J Med       Date:  2021-06-26       Impact factor: 91.245

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