Skipping along: an exon skipping therapy shows promise for Duchenne muscular dystrophy

Affiliations

Comment

A K Hawkins. Clin Genet. 2011 Nov.

. 2011 Nov;80(5):424-5.

doi: 10.1111/j.1399-0004.2011.01769.x. Epub 2011 Sep 12.

¹ Department of Medical Genetics, Centre for Molecular Medicine and Therapeutics, University of British Columbia, Vancouver, Canada. alicehaw@exchange.ubc.ca

No abstract available

Comment on

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study.
Cirak S, Arechavala-Gomeza V, Guglieri M, Feng L, Torelli S, Anthony K, Abbs S, Garralda ME, Bourke J, Wells DJ, Dickson G, Wood MJ, Wilton SD, Straub V, Kole R, Shrewsbury SB, Sewry C, Morgan JE, Bushby K, Muntoni F. Cirak S, et al. Lancet. 2011 Aug 13;378(9791):595-605. doi: 10.1016/S0140-6736(11)60756-3. Epub 2011 Jul 23. Lancet. 2011. PMID: 21784508 Free PMC article. Clinical Trial.