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-Reference-Cited by-同舟云学术

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

Published:2009-10 Issue:10 Volume:8 Page:918-928
ISSN:1474-4422
Container-title:The Lancet Neurology
language:en
Short-container-title:The Lancet Neurology

Author:

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

Publisher

Elsevier BV

Subject

Neurology (clinical)

Reference42 articles.

1. Impact of nasal ventilation on survival in hypercapnic Duchenne muscular dystrophy;Simonds;Thorax,1998

2. Survival in Duchenne muscular dystrophy: improvements in life expectancy since 1967 and the impact of home nocturnal ventilation;Eagle;Neuromuscul Disord,2002

3. Dystrophin: the protein product of the Duchenne muscular dystrophy locus;Hoffman;Cell,1987

4. A quantitative ELISA for dystrophin;Morris;J Immunol Methods,1993

5. The molecular basis for Duchenne versus Becker muscular dystrophy: correlation of severity with type of deletion;Koenig;Am J Hum Genet,1989

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