Neuromuscular disease articles within Gene Therapy

Featured

• Review | 28 July 2017

  Management of neuromuscular diseases and spinal muscular atrophy in Latin America

  • S Monges
  •  & A L Rosa

• Review | 31 May 2017

  Therapeutic approaches for spinal muscular atrophy (SMA)

  • M Scoto
  • , R S Finkel
  •  & F Muntoni

• Review | 31 May 2017

  The SMA Trust: the role of a disease-focused research charity in developing treatments for SMA

  • V Christie-Brown
  • , J Mitchell
  •  & K Talbot

• Review | 30 May 2017

  Developmental regulation of SMN expression: pathophysiological implications and perspectives for therapy development in spinal muscular atrophy

  • S Jablonka
  •  & M Sendtner

• Original Article
  30 May 2013 | Open Access

  Effect of vascular endothelial growth factor gene therapy on post-traumatic peripheral nerve regeneration and denervation-related muscle atrophy

  • S Moimas
  • , F Novati
  •  & S Raimondo

• Original Article | 03 May 2012

  MRI roadmap-guided transendocardial delivery of exon-skipping recombinant adeno-associated virus restores dystrophin expression in a canine model of Duchenne muscular dystrophy

  • I M Barbash
  • , S Cecchini
  •  & R M Kotin

• Review | 09 February 2012

  Progress in gene therapy of dystrophic heart disease

  • Y Lai
  •  & D Duan

• Short Communication
  26 January 2012 | Open Access

  AAV-based shRNA silencing of NF-κB ameliorates muscle pathologies in mdx mice

  • Q Yang
  • , Y Tang
  •  & B Wang