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Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease

Kim, Jinkuk; Hu, C.; El Achkar, C. Moufawad; Black, L. E.; Douville, J.; Larson, A.; Pendergast, M. K.; et al, NEW ENGLAND JOURNAL OF MEDICINE, v.381, no.17, pp.1644 - 1652, 2019-10

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