Seamless Genetic Conversion of SMN2 to SMN1 via CRISPR/Cpf1 and Single-Stranded Oligodeoxynucleotides in Spinal Muscular Atrophy Patient-Specific Induced Pluripotent Stem Cells

Published 2018 View Full Article

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Journal

HUMAN GENE THERAPY

Volume -, Issue -, Pages -

Publisher

Mary Ann Liebert Inc

Online

2018-03-30

DOI

10.1089/hum.2017.255

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HUMAN GENE THERAPY

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