Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

BACKGROUND: Mutations that disrupt the open reading frame and prevent full translation of DMD, the gene that encodes dystrophin, underlie the fatal X-linked disease Duchenne muscular dystrophy. Oligonucleotides targeted to splicing elements (splice switching oligonucleotides) in DMD pre-mRNA can lea...

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Detaylı Bibliyografya
Asıl Yazarlar: Kinali, Maria, Arechavala-Gomeza, Virginia, Feng, Lucy, Cirak, Sebahattin, Hunt, David, Adkin, Carl, Guglieri, Michela, Ashton, Emma, Abbs, Stephen, Nihoyannopoulos, Petros, Garralda, Maria Elena, Rutherford, Mary, Mcculley, Caroline, Popplewell, Linda, Graham, Ian R, Dickson, George, Wood, Matthew JA, Wells, Dominic J, Wilton, Steve D, Kole, Ryszard, Straub, Volker, Bushby, Kate, Sewry, Caroline, Morgan, Jennifer E, Muntoni, Francesco
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: Lancet Pub. Group 2009
Konular:
Fast track — Articles
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC2755039/
https://ncbi.nlm.nih.gov/pubmed/19713152
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/S1474-4422(09)70211-X
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