Thibaut Marais

GDF5 as a Multimodal Protector of the Motor Unit in Amyotrophic Lateral Sclerosis

Aly Bourguiba, Sonia Pezet, Massiré Traoré, Christel Gentil et autres

fr, it (code pays fourni par la source)

Novel intravenous AAV gene therapy for mucopolysaccharidosis type IIIA and IIIB in mouse and canine model of the pathology – Towards clinical translation

Françoise Piguet, Rafael Alonso, Elena Gaia Banchi, Thibaut Marais et autres

Pre-clinical evaluation of a gene therapy candidate for SOD1 -ALS shows improved survival and signs of inflammation in the CNS of treated mice

Sonia Pezet, Jennifer Hua, Thibaut Marais, Marine Delamare et autres

fr, it (code pays fourni par la source)

177P Effect of gene therapy on the epigenetic and transcriptional stability of SMA

Piera Smeriglio, Fiorella C. Grandi, A. Arnould, Sonia Pezet et autres

Muscle regeneration affects Adeno Associated Virus 1 mediated transgene transcription

Amédée Mollard, Cécile Peccate, Anne Forand, Julie Chassagne et autres

fr (code pays fourni par la source)

Promoterless Gene Targeting Approach Combined to CRISPR/Cas9 Efficiently Corrects Hemophilia B Phenotype in Neonatal Mice

Michela Lisjak, Alessia De Caneva, Thibaut Marais, Elena Barbon et autres

it, fr, il, us (code pays fourni par la source)

The non-muscle ADF/cofilin-1 controls sarcomeric actin filament integrity and force production in striated muscle laminopathies

Nicolas Vignier, Maria Chatzifrangkeskou, Luca Pinton, Hugo Wioland et autres

fr, gb, us (code pays fourni par la source)

Systemic Treatment of Fabry Disease Using a Novel AAV9 Vector Expressing α-Galactosidase A

Maria Grazia Biferi, Mathilde Cohen-Tannoudji, Andrea García-Silva, Olga Souto-Rodríguez et autres

fr, es (code pays fourni par la source)

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

Sahar Elouej, Marine Delamare, M Cappella, Mathilde Cohen-Tannoudji et autres

fr (code pays fourni par la source)

HEREDITARY NEUROPATHIES & ALS

Sahar Elouej, M. Delamare, M Cappella, Mathilde Cohen-Tannoudji et autres

fr (code pays fourni par la source)

Analysis of off-target effects of Antisense Sequence inducing Exon Skipping in SOD1-linked Amyotrophic Lateral Sclerosis

Sahar Elouej, Marine Delamare, M Cappella, Mathilde Cohen-Tannoudji et autres

fr (code pays fourni par la source)

Activation of sarcolipin expression and altered calcium cycling in LMNA cardiomyopathy

Blanca Morales Rodríguez, Alejandro Domínguez‐Rodríguez, Jean‐Pierre Bénitah, Florence Lefebvre et autres

fr (code pays fourni par la source)