A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse

P.N. Porensky, Chalermchai Mitrpant, V.L. Mcgovern, A.K. Bevan, K.D. Foust, B.K. Kaspar, Steve Wilton, A.H. Burghes

Research output: Contribution to journalArticlepeer-review

208 Citations (Scopus)
Original languageEnglish
Pages (from-to)1625-1638
JournalHuman Molecular Genetics
Issue number7
Publication statusPublished - 2012

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