A short antisense oligonucleotide ameliorates symptoms of severe mouse models of spinal muscular atrophy

Jeffrey M. Keil, Joonbae Seo, Matthew D. Howell, Walter H. Hsu, Ravindra N. Singh*, Christine J. DiDonato

*Corresponding author for this work

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Medicine and Dentistry

Biochemistry, Genetics and Molecular Biology

Pharmacology, Toxicology and Pharmaceutical Science