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Skipping along: an exon skipping therapy shows promise for Duchenne muscular dystrophy
Author(s) -
Hawkins AK
Publication year - 2011
Publication title -
clinical genetics
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.543
H-Index - 102
eISSN - 1399-0004
pISSN - 0009-9163
DOI - 10.1111/j.1399-0004.2011.01769.x
Subject(s) - duchenne muscular dystrophy , exon skipping , morpholino , medicine , dystrophin , muscular dystrophy , exon , genetics , biology , alternative splicing , zebrafish , gene
References 1. Cirak S , Arechavala‐Gomeza V , Guglieri M et al. 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 . Lancet 2011 : 378 ( 9791 ): 595 – 605.2. Bushby K , Finkel R , Birnkrant DJ et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management . Lancet Neurol 2010 : 9 ( 1 ): 77 – 93.3. Nakamura A , Takeda S. Exon‐skipping therapy for Duchenne muscular dystrophy . Lancet 2011 : 378 ( 9791 ): 546 – 547. 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, et al. (2011) The Lancet 378 (9791): 595–605