Author

Ophélie Vacca

InsermORCID 0000-0001-9281-843XUnclaimed ORCID profile · follow to get notified of new preprints

Biology Disciplines

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Signature Assays, Organisms & Methods

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Organism:mouse· 3 Approach:experimental assay· 2 Research Focus:cell type annotation· 1 Research Focus:differential expression· 1 Research Focus:drug response / drug discovery· 1 Assay:spatial transcriptomics· 1 Approach:statistical model· 1

Frequent Co-Authors

Aurélie GoyenvalleiD· 3 shared Mathilde DoisyiD· 3 shared Amel Saoudi· 2 shared Cathy NagyiD· 2 shared Cyrille VaillendiD· 2 shared Olivier Le CoziD· 2 shared Xaysongkhame PhongsavanhiD· 2 shared Ahmed Essam MahfouziD· 1 shared

Activity by Year

2025-2026 · peak 2/yr

Co-Author Collaboration Network

10 top collaborators · 30 mutual co-author links

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3 shared papersNeuroscience

Aurélie GoyenvalleORCID

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3 papers

Recent·Most cited

NeurosciencebioRxivPreprintOct 15, 2026

Neonatal Delivery of Exon Skipping Therapeutics Maximizes Brain Dystrophin Rescue in the Mdx52 Mouse Model of Duchenne Muscular Dystrophy

Ophélie Vacca, Mathilde Doisy, Cathy Nagy…·Aurélie Goyenvalle

The paper shows that neonatal intracerebroventricular delivery of scAAV9-U7-Ex51 provides sustained exon skipping and dystrophin restoration in the brain of mdx52 mice, outperforming systemic or intracerebroventricular ASO delivery.

DOI 10.64898/2026.10.09.757591

mouseexperimental assay

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NeuroscienceiSciencePeer-reviewedJul 23, 2026

Evaluating exon-skipping therapies targeting the central nervous system in Duchenne muscular dystrophy using high-resolution spatial transcriptomics

Qirong Mao, Alireza Ahmadi, Sharon I. de Vries…·Pietro Spitali

This study uses spatial transcriptomics to evaluate exon-skipping therapies for DMD in the CNS, showing U7ex51 effectively restores full-length dystrophin isoforms in neurons.

DOI 10.1016/j.isci.2026.116906

mousespatial transcriptomics

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NeuroscienceMolecular Therapy — Nucleic AcidsPeer-reviewedNov 20, 2025

Ineffective behavioral rescue despite partial brain Dp427 restoration by AAV9-U7-mediated exon 51 skipping in mdx52 mice

Ophélie Vacca, Amel Saoudi, Mathilde Doisy…·Aurélie Goyenvalle

This study evaluates AAV9-U7-mediated exon 51 skipping in mdx52 mice to restore Dp427 and improve behavioral outcomes, finding limited Dp427 restoration and no behavioral improvement.

4 citations

mouseexperimental assay

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