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Journal of Neuromuscular Diseases|July 30, 2025
Beyond muscle: Delivering RNA therapeutics to the CNS in Duchenne muscular dystrophyOphélie Vacca, Cathy Nagy, Aurélie Goyenvalle
Investigative Ophthalmology & Visual Science|November 9, 2017
The 3D Retinal Capillary Circulation in Pigs Reveals a Predominant Serial OrganizationStéphane Fouquet, Ophélie Vacca, Florian Sennlaub, et al.
Nucleic Acid Therapeutics|July 24, 2024
Levels of Exon-Skipping Are Not Artificially Overestimated Because of the Increased Affinity of Tricyclo-DNA-Modified Antisense Oligonucleotides to the Target DMD ExonMathilde Doisy, Ophélie Vacca, Amel Saoudi, et al.
International Journal of Molecular Sciences|October 27, 2022
Abnormal Expression of Synaptic and Extrasynaptic GABAA Receptor Subunits in the Dystrophin-Deficient mdx MouseFaouzi Zarrouki, Sébastien Goutal, Ophélie Vacca, et al.
Journal of Visualized Experiments : Jove|May 5, 2015
Using Adeno-associated Virus as a Tool to Study Retinal Barriers in DiseaseOphélie Vacca, Brahim El Mathari, Marie Darche, et al.
Cells|April 26, 2024
AAV-Mediated Restoration of Dystrophin-Dp71 in the Brain of Dp71-Null Mice: Molecular, Cellular and Behavioral OutcomesOphélie Vacca, Faouzi Zarrouki, Charlotte Izabelle, et al.
Glia|January 3, 2014
AAV-mediated gene delivery in Dp71-null mouse model with compromised barriersOphélie Vacca, Marie Darche, David V Schaffer, et al.
Molecular Neurobiology|January 28, 2017
Dystrophin Dp71 Isoforms Are Differentially Expressed in the Mouse Brain and Retina: Report of New Alternative Splicing and a Novel Nomenclature for Dp71 IsoformsJorge Aragón, Mayram González-Reyes, José Romo-Yáñez, et al.
Molecular Therapy. Nucleic Acids|December 23, 2025
Ineffective behavioral rescue despite partial brain Dp427 restoration by AAV9-U7-mediated exon 51 skipping in mdx52 miceOphélie Vacca, Amel Saoudi, Mathilde Doisy, et al.
Molecular Vision|December 10, 2019
Evidence of the involvement of dystrophin Dp71 in corneal angiogenesisGabriella Ortiz, Ophélie Vacca, Romain Bénard, et al.
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