VIRGINIA
ARECHAVALA GOMEZA
Leiden University Medical Center
Leiden, HolandaPublikationen in Zusammenarbeit mit Forschern von Leiden University Medical Center (13)
2023
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Considerations in the Preclinical Assessment of the Safety of Antisense Oligonucleotides
Nucleic acid therapeutics, Vol. 33, Núm. 1, pp. 1-16
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Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi
Molecular Therapy Nucleic Acids, Vol. 34
2021
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Delivery of oligonucleotide-based therapeutics: challenges and opportunities
EMBO Molecular Medicine, Vol. 13, Núm. 4
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Sharing 'Negative' Results in Neuromuscular Research: A Positive Experience
Journal of Neuromuscular Diseases
2019
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Report of a TREAT-NMD/World duchenne organisation meeting on dystrophin quantification methodology
Journal of Neuromuscular Diseases
2018
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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
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Why dystrophin quantification is key in the eteplirsen saga
Nature reviews. Neurology
2017
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Delivery is key: lessons learnt from developing splice-switching antisense therapies
EMBO Molecular Medicine, Vol. 9, Núm. 5, pp. 545-557
2016
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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
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Splicing modulation therapy in the treatment of genetic diseases
Application of Clinical Genetics, Vol. 7, pp. 245-252
2010
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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
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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