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

  1. Lookup NU author(s)
  2. Dr Michela Guglieri
  3. Professor Volker Straub
  4. Professor Katherine Bushby
Author(s)Kinali M, Arechavala-Gomeza V, Feng L, Cirak S, Hunt D, Adkin C, Guglieri M, Ashton E, Abbs S, Nihoyannopoulos P, Garralda M, Rutherford M, Mcculley C, Popplewell L, Graham I, Dickson G, Wood M, Wells D, Wilton S, Kole R, Straub V, Bushby K, Sewry C, Morgan J, Muntoni F
Publication type Article
JournalThe Lancet Neurology
ISSN (print)1474-4422
ISSN (electronic)1474-4465
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PublisherThe Lancet Publishing Group
PubMed id19713152
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