Aurélie Goyenvalle

Inserm — France

Biochemistry, Genetics and Molecular Biology · Life Sciences

29h-index3.7kcitations125works2.52yr avg
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Research Topics

Muscle Physiology and Disorders(79), RNA Interference and Gene Delivery(44), CRISPR and Genetic Engineering(27), Neurogenetic and Muscular Disorders Research(24), RNA Research and Splicing(23)

Publications125 total

Improving angiogenesis ameliorates the efficacy of ASO-based exon skipping for the treatment of Duchenne muscular dystrophy
Molecular Therapy — Nucleic Acids·2026Open Access
The complexity of dystrophin transcription and processing: implications of transcript imbalance on dystrophin gene targeting strategies
Neuromuscular Disorders·2025
Ineffective behavioral rescue despite partial brain Dp427 restoration by AAV9-U7-mediated exon 51 skipping in mdx52 mice
Molecular Therapy — Nucleic Acids·2025· 1 citedOpen Access
Targeting a Pathogenic Variant Creating an Upstream AUG in the <i>ENG</i> 5′ Untranslated Region with Antisense Oligonucleotides Fails to Restore Protein Expression
Nucleic Acid Therapeutics·2025· 1 cited
Evaluating exon-skipping therapies targeting the central nervous system in Duchenne muscular dystrophy using high-resolution spatial transcriptomics
2025Open Access
Ineffective behavioral rescue despite partial brain Dp427 restoration by AAV9-U7-mediated exon 51 skipping in <i>mdx52</i> mice
2025
287P3D tissue engineered skeletal muscle to model dystrophinopathies and determine the efficacy of exon skip therapies
Neuromuscular Disorders·2025
Improving angiogenesis ameliorates the efficacy of ASO-based exon-skipping for the treatment of Duchenne muscular dystrophy
2025Open Access
Rosalind Franklin Society Proudly Announces the 2024 Award Recipient for <i>Nucleic Acid Therapeutics</i>
Nucleic Acid Therapeutics·2025
Beyond muscle: Delivering RNA therapeutics to the CNS in Duchenne muscular dystrophy
Journal of Neuromuscular Diseases·2025· 1 citedOpen Access
Overlapping upstream ORFs ending at c.125 lead to reduced Endoglin, contributing to Hereditary Hemorrhagic Telangiectasia
Communications Biology·2025· 1 citedOpen Access
A genome-wide CRISPR screen unveils the endosomal maturation protein WDR91 as a promoter of productive ASO activity in melanoma
Molecular Therapy — Nucleic Acids·2025· 3 citedOpen Access
Overlapping upstream ORFs ending at c.125 lead to reduced Endoglin, contributing to Hereditary Hemorrhagic Telangiectasia
2025Open Access
Valproic Acid Improves Antisense-Mediated Exon-Skipping Efficacy in mdx Mice
International Journal of Molecular Sciences·2025· 2 citedOpen Access
Combined 20-Hydroxyecdysone and Antisense-Mediated Exon Skipping Improve Functional Outcomes in a Mouse Model of Duchenne Muscular Dystrophy
Nucleic Acid Therapeutics·2025· 3 citedOpen Access
Duchenne muscular dystrophy: recent insights in brain related comorbidities
Nature Communications·2025· 31 citedOpen Access
Use of Tricyclo-DNA Antisense Oligonucleotides for Exon-Skipping
Methods in molecular biology·2025
Impact of distinct dystrophin gene mutations on behavioral phenotypes of Duchenne muscular dystrophy
Disease Models & Mechanisms·2024· 6 citedOpen Access
300P Brain involvement in Duchenne muscular dystrophy (BIND)
Neuromuscular Disorders·2024
306P Evaluating genetic therapies targeting the central nervous system in Duchenne muscular dystrophy using high-resolution spatial transcriptomics
Neuromuscular Disorders·2024
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Frequent Co-authors

Luis Garcı́a(25), Kay E. Davies(11), Cyrille Vaillend(11), Lucía Echevarría(7), Arran Babbs(7), Faouzi Zarrouki(7), Thomas Tensorer(7), Olivier Danos(6), Christian J. Leumann(6), Philippine Aupy(6), Karima Relizani(6), Annemieke Aartsma‐Rus(5), Graziella Griffith(5), Adeline Vulin(4), Virginia Arechavala‐Gomeza(4), Matthew J. A. Wood(4), Stéphanie Lorain(4), Taavi Lehto(3), Willeke van Roon‐Mom(3), Samir EL Andaloussi(3)