Publicaciones en colaboración con investigadores/as de Imperial College London (4)

2011

  1. Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: An open-label, phase 2, dose-escalation study

    The Lancet, Vol. 378, Núm. 9791, pp. 595-605

2010

  1. Immunohistological intensity measurements as a tool to assess sarcolemma-associated protein expression

    Neuropathology and Applied Neurobiology, Vol. 36, Núm. 4, pp. 265-274

2009

  1. 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

    The Lancet Neurology, Vol. 8, Núm. 10, pp. 918-928

2007

  1. Comparative analysis of antisense oligonucleotide sequences for targeted skipping of exon 51 during dystrophin pre-mRNA splicing in human muscle

    Human Gene Therapy, Vol. 18, Núm. 9, pp. 798-810