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Proceedings of the National Academy of Sciences of the United States of America|December 20, 2000
Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouseC J Mann, K Honeyman, A J Cheng, et al.
International Journal of Molecular Sciences|April 12, 2022
Antisense Oligonucleotide Induction of the hnRNPA1b Isoform Affects Pre-mRNA Splicing of SMN2 in SMA Type I FibroblastsJarichad Toosaranont, Sukanya Ruschadaariyachat, Warasinee Mujchariyakul, et al.
Human Gene Therapy|January 24, 2013
A novel morpholino oligomer targeting ISS-N1 improves rescue of severe spinal muscular atrophy transgenic miceHaiyan Zhou, Narinder Janghra, Chalermchai Mitrpant, et al.
Nature Genetics|January 1, 1995
A mutation in the alpha tropomyosin gene TPM3 associated with autosomal dominant nemaline myopathyN G Laing, S D Wilton, P A Akkari, et al.
Human Molecular Genetics|December 22, 2011
A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mousePaul N Porensky, Chalermchai Mitrpant, Vicki L McGovern, et al.
American Journal of Human Genetics|February 1, 1995
Autosomal dominant distal myopathy: linkage to chromosome 14N G Laing, B A Laing, C Meredith, et al.
Nature Medicine|July 9, 2003
Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouseQi Long Lu, Christopher J Mann, Fang Lou, et al.
Neuromuscular Disorders : NMD|March 14, 2000
Severe gamma-sarcoglycanopathy caused by a novel missense mutation and a large deletionK J Nowak, P Walsh, R L Jacob, et al.
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