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

  • Maria Kinali
  • , Virginia Arechavala-Gomeza
  • , Lucy Feng
  • , Sebahattin Cirak
  • , David Hunt
  • , Carl Adkin
  • , Michela Guglieri
  • , Emma Ashton
  • , Stephen Abbs
  • , Petros Nihoyannopoulos
  • , Maria Elena Garralda
  • , Mary Rutherford
  • , Caroline McCulley
  • , Linda Popplewell
  • , Ian R Graham
  • , George Dickson
  • , Matthew J A Wood
  • , Dominic J Wells
  • , Steve D Wilton
  • , Ryszard Kole
    • Volker Straub, Kate Bushby, Caroline Sewry, Jennifer E Morgan, Francesco Muntoni

Research output: Contribution to journal › Article › peer-review

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Medicine and Dentistry

Neuroscience

Biochemistry, Genetics and Molecular Biology