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Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease
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The New England Journal of Medicine
From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine.
Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease
N. Engl. J. Med 2019 Oct 09;[EPub Ahead of Print], J Kim, C Hu, C Moufawad El Achkar, LE Black, J Douville, A Larson, MK Pendergast, SF Goldkind, EA Lee, A Kuniholm, A Soucy, J Vaze, NR Belur, K Fredriksen, I Stojkovska, A Tsytsykova, M Armant, RL DiDonato, J Choi, L Cornelissen, LM Pereira, EF Augustine, CA Genetti, K Dies, B Barton, L Williams, BD Goodlett, BL Riley, A Pasternak, ER Berry, KA Pflock, S Chu, C Reed, K Tyndall, PB Agrawal, AH Beggs, PE Grant, DK Urion, RO Snyder, SE Waisbren, A Poduri, PJ Park, A Patterson, A Biffi, JR Mazzulli, O Bodamer, CB Berde, TW YuFrom MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine.