Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease

Jin‐Kuk Kim(Hanyang University), Timothy W. Yu(Boston Children's Hospital), Eunjung A. Lee(Broad Institute), Myriam Armant(Dana-Farber/Boston Children's Cancer and Blood Disorders Center), Richard O. Snyder(Inserm), Annapurna Poduri(Broad Institute), Alessandra Biffi(University of Padua), Bobbie Riley(Boston Children's Hospital), Peter J. Park(Harvard University), Christelle Moufawad El Achkar(Harvard University), Stephen J. Chu(Unknown), Laura Cornelissen(Harvard University), Kelly A. Pflock(Anna Needs Neuroblastoma Answers), Alla V. Tsytsykova(Boston Children's Hospital), Nandkishore R. Belur(Northwestern University), Chunguang Hu(Institut thématique Génétique, génomique et bioinformatique), Jaejoon Choi(Institut thématique Génétique, génomique et bioinformatique), Austin Larson(Children's Hospital Colorado), Ashley Kuniholm(Boston Children's Hospital), Mary K. Pendergast(Anna Needs Neuroblastoma Answers), Kristina Fredriksen(Northwestern University), Alan H. Beggs(Boston Children's Hospital), Charles B. Berde(Boston Children's Hospital), Lauren E. Black(Anna Needs Neuroblastoma Answers), Kimberly Tyndall(Anna Needs Neuroblastoma Answers), Luis M. Pereira(Boston Children's Hospital), P. Ellen Grant(Boston Children's Hospital), Erika F. Augustine(University of Rochester Medical Center), Sara F. Goldkind(Anna Needs Neuroblastoma Answers), Emily R. Berry(Anna Needs Neuroblastoma Answers), Julie Douville(Charles River Laboratories (Netherlands)), Renata L. DiDonato(Institut thématique Génétique, génomique et bioinformatique), Chantal Reed(Anna Needs Neuroblastoma Answers), Aubrie Soucy(Institut thématique Génétique, génomique et bioinformatique), Benjamin D. Goodlett(Boston Children's Hospital), Lucinda Williams(Boston Children's Hospital), Joseph R. Mazzulli(Harvard University), Brenda Barton(Boston Children's Hospital), Olaf A. Bodamer(Universitätsklinik für Kinder und Jugendpsychiatrie), Pankaj B. Agrawal(Post Graduate Institute of Medical Education and Research), David K. Urion(Harvard University), Iva Stojkovska(Northwestern University), Amy Pasternak(Anna Needs Neuroblastoma Answers), Casie A. Genetti(Boston Children's Hospital), Al Patterson(Harvard University), Kira A. Dies(Boston Children's Hospital), Susan E. Waisbren(Boston Children's Hospital), Jai Vaze(Institut thématique Génétique, génomique et bioinformatique)
New England Journal of Medicine
October 9, 2019
Cited by 808


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