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STUDY OF POTENTIAL OFF-TARGET CANDIDATE SITES FOR ANTISENSE SEQUENCES INDUCING EXON SKIPPING IN SOD1-LINKED AMYOTROPHIC LATERAL SCLEROSIS

Autor: Elouej, Sahar, Marine, Delamare, Cappella, Marisa, Cohen-Tannoudji, Mathilde, Astord, Stéphanie, Marais, Thibaut, Giroux, Benoit, PEZET, Sonia, Biferi, Maria

Publikováno v: JR6 FILSLAN-ARSLA
JR6 FILSLAN-ARSLA, Oct 2020, Online, France

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=od______3515::86da70e8b8caede1dfb350100a469b66
https://hal.science/hal-04002468

AAV9-Mediated Expression of SMN Restricted to Neurons Does Not Rescue the Spinal Muscular Atrophy Phenotype in Mice

Autor: Besse, Aurore, Astord, Stephanie, Marais, Thibaut, Roda, Marianne, Giroux, Benoit, Lejeune, François-Xavier, Relaix, Frederic, Smeriglio, Piera, Barkats, Martine, Biferi, Maria Grazia

Publikováno v: Molecular Therapy
Molecular Therapy, Cell Press, 2020, 28 (8), pp.1887-1901. ⟨10.1016/j.ymthe.2020.05.011⟩
Molecular Therapy, 2020, 28 (8), pp.1887-1901. ⟨10.1016/j.ymthe.2020.05.011⟩

Spinal muscular atrophy (SMA) is a neuromuscular disease mainly caused by mutations or deletions in the survival of motor neuron 1 (SMN1) gene and characterized by the degeneration of motor neurons and progressive muscle weakness. A viable therapeuti

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=pmid_dedup__::c8757ad150ccee3a8fecb2e5f7ff4d63
https://www.hal.inserm.fr/inserm-03130706/document

AAV9- mediated expression of SMN restricted to neurons does not rescue the spinal muscular atrophy phenotype

Autor: Smeriglio, Piera, Besse, Aurore, Astord, Stéphanie, Giroux, Benoit, Marais, Thibaut, Relaix, Frédéric, Barkats, Martine, Biferi, Maria

Publikováno v: 2e congrès scientifique international de SMA Europe
2e congrès scientifique international de SMA Europe, Feb 2020, EVRY, France

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=dedup_wf_001::0781654a0420bfbb3f9d5b7fb70c29dc
https://hal.science/hal-04002484

AAV-mediated expression of antisense oligonucleotides for the treatment of C9orf72-ALS

Autor: Cappella, Marisa, Marais, Thibaut, Astord, Stéphanie, Giroux, Benoit, Bigot, Anne, Mouly, Vincent, Lefebvre Omar, Cynthia, Bohl, Delphine, Biferi, Maria

Publikováno v: 27th ESGCT Congress
27th ESGCT Congress, Oct 2019, BARCELONE, Spain

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=od______3515::a3eb67ec6c2892653d7bf24a917dc77c
https://hal.science/hal-04002234