AAV-Mediated Restoration of Dystrophin-Dp71 in the Brain of Dp71-Null Mice: Molecular, Cellular and Behavioral Outcomes

Autor: Ophélie Vacca, Faouzi Zarrouki, Charlotte Izabelle, Mehdi Belmaati Cherkaoui, Alvaro Rendon, Deniz Dalkara, Cyrille Vaillend

Publikováno v: Cells, Vol 13, Iss 8, p 718 (2024)

A deficiency in the shortest dystrophin-gene product, Dp71, is a pivotal aggravating factor for intellectual disabilities in Duchenne muscular dystrophy (DMD). Recent advances in preclinical research have achieved some success in compensating both mu

Externí odkaz: https://doaj.org/article/eca27be8b77145bdab86d84d8f2672b5

Long-Term Efficacy of AAV9-U7snRNA-Mediated Exon 51 Skipping in mdx52 Mice

Autor: Philippine Aupy, Faouzi Zarrouki, Quentin Sandro, Cécile Gastaldi, Pierre-Olivier Buclez, Kamel Mamchaoui, Luis Garcia, Cyrille Vaillend, Aurélie Goyenvalle

Publikováno v: Molecular Therapy: Methods & Clinical Development, Vol 17, Iss , Pp 1037-1047 (2020)

Gene therapy and antisense approaches hold promise for the treatment of Duchenne muscular dystrophy (DMD). The advantages of both therapeutic strategies can be combined by vectorizing antisense sequences into an adeno-associated virus (AAV) vector. W

Externí odkaz: https://doaj.org/article/91dce3aef9fb4e71bff890b0fec6ee34

Efficacy and Safety Profile of Tricyclo-DNA Antisense Oligonucleotides in Duchenne Muscular Dystrophy Mouse Model

Autor: Karima Relizani, Graziella Griffith, Lucía Echevarría, Faouzi Zarrouki, Patricia Facchinetti, Cyrille Vaillend, Christian Leumann, Luis Garcia, Aurélie Goyenvalle

Publikováno v: Molecular Therapy: Nucleic Acids, Vol 8, Iss , Pp 144-157 (2017)

Antisense oligonucleotides (AONs) hold promise for therapeutic splice-switching correction in many genetic diseases. However, despite advances in AON chemistry and design, systemic use of AONs is limited due to poor tissue uptake and sufficient thera

Externí odkaz: https://doaj.org/article/1d94d53beb4a47d8a7a1a68df902a18f

Abnormal Expression of Synaptic and Extrasynaptic GABA

Autor: Faouzi, Zarrouki, Sébastien, Goutal, Ophélie, Vacca, Luis, Garcia, Nicolas, Tournier, Aurélie, Goyenvalle, Cyrille, Vaillend

Publikováno v: International journal of molecular sciences. 23(20)

Duchenne muscular dystrophy (DMD) is a neurodevelopmental disorder primarily caused by the loss of the full-length Dp427 dystrophin in both muscle and brain. The basis of the central comorbidities in DMD is unclear. Brain dystrophin plays a role in t

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=pmid________::e7de221d37128edf0b5fbe0ecee51ccb
https://pubmed.ncbi.nlm.nih.gov/36293496

Emotional behavior and brain anatomy of the mdx52 mouse model of Duchenne muscular dystrophy

Autor: Cyrille Vaillend, Amel Saoudi, Catherine Sebrié, Aurélie Goyenvalle, Faouzi Zarrouki, Charlotte Izabelle

Publikováno v: Disease Models & Mechanisms
Disease Models & Mechanisms, 2021, 14 (9), pp.dmm049028. ⟨10.1242/dmm.049028⟩
Disease Models & Mechanisms, Cambridge Company of Biologists, 2021, 14 (9), pp.dmm049028. ⟨10.1242/dmm.049028⟩
Disease Models & Mechanisms, Vol 14, Iss 9 (2021)
article-version (VoR) Version of Record

The exon-52-deleted mdx52 mouse is a critical model of Duchenne muscular dystrophy (DMD), as it features a deletion in a hotspot region of the DMD gene, frequently mutated in patients. Deletion of exon 52 impedes expression of several brain dystrophi

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::08d925b2f297a821e724b3b33ad4221e
https://hal.science/hal-03407894/document