Publicaciones en colaboración con investigadores/as de Leiden University Medical Center (13)

2023

  1. Considerations in the Preclinical Assessment of the Safety of Antisense Oligonucleotides

    Nucleic acid therapeutics, Vol. 33, Núm. 1, pp. 1-16

  2. Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi

    Molecular Therapy Nucleic Acids, Vol. 34

2021

  1. Delivery of oligonucleotide-based therapeutics: challenges and opportunities

    EMBO Molecular Medicine, Vol. 13, Núm. 4

  2. Sharing 'Negative' Results in Neuromuscular Research: A Positive Experience

    Journal of Neuromuscular Diseases

2019

  1. Report of a TREAT-NMD/World duchenne organisation meeting on dystrophin quantification methodology

    Journal of Neuromuscular Diseases

2018

  1. A multicenter comparison of quantification methods for antisense oligonucleotide-induced DMD exon 51 skipping in Duchenne muscular dystrophy cell cultures

    PLoS ONE, Vol. 13, Núm. 10

  2. Why dystrophin quantification is key in the eteplirsen saga

    Nature reviews. Neurology

2017

  1. Delivery is key: lessons learnt from developing splice-switching antisense therapies

    EMBO Molecular Medicine, Vol. 9, Núm. 5, pp. 545-557

2016

  1. Stakeholder cooperation to overcome challenges in orphan medicine development: The example of Duchenne muscular dystrophy

    The Lancet Neurology, Vol. 15, Núm. 8, pp. 882-890

2014

  1. Splicing modulation therapy in the treatment of genetic diseases

    Application of Clinical Genetics, Vol. 7, pp. 245-252

2011

  1. Dystrophin quantification and clinical correlations in Becker muscular dystrophy: Implications for clinical trials

    Brain

2010

  1. Comparative analysis of antisense oligonucleotide sequences targeting exon 53 of the human DMD gene: Implications for future clinical trials

    Neuromuscular Disorders, Vol. 20, Núm. 2, pp. 102-110

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