| Gene therapy for ALS—a perspective M Cappella, C Ciotti, M Cohen-Tannoudji, MG Biferi International journal of molecular sciences 20 (18), 4388, 2019 | 90 | 2019 |
| CRISPR/Cas9-mediated deletion of CTG expansions recovers normal phenotype in myogenic cells derived from myotonic dystrophy 1 patients C Provenzano, M Cappella, R Valaperta, R Cardani, G Meola, F Martelli, ... Molecular Therapy-Nucleic Acids 9, 337-348, 2017 | 71 | 2017 |
| MicroRNA-222 regulates muscle alternative splicing through Rbm24 during differentiation of skeletal muscle cells B Cardinali, M Cappella, C Provenzano, JM Garcia-Manteiga, ... Cell death & disease 7 (2), e2086-e2086, 2016 | 50 | 2016 |
| Beyond the traditional clinical trials for amyotrophic lateral sclerosis and the future impact of gene therapy M Cappella, PF Pradat, G Querin, MG Biferi Journal of Neuromuscular Diseases 8 (1), 25-38, 2021 | 33 | 2021 |
| High-throughput analysis of the RNA-induced silencing complex in myotonic dystrophy type 1 patients identifies the dysregulation of miR-29c and its target ASB2 M Cappella, A Perfetti, B Cardinali, JM Garcia-Manteiga, M Carrara, ... Cell death & disease 9 (7), 729, 2018 | 22 | 2018 |
| The potential of induced pluripotent stem cells to test gene therapy approaches for neuromuscular and motor neuron disorders M Cappella, S Elouej, MG Biferi Frontiers in Cell and Developmental Biology 9, 662837, 2021 | 7 | 2021 |
| Activation of tyrosine phosphorylation signaling in erythrocytes of patients with Alzheimer’s disease C Mallozzi, A Crestini, C d'Amore, P Piscopo, M Cappella, F Perrone, ... Neuroscience 433, 36-41, 2020 | 7 | 2020 |
| Antisense sequences for treating amyotrophic lateral sclerosis M Biferi, M Cappella, M Barkats US Patent App. 17/917,953, 2023 | | 2023 |
| A new AAV-mediated gene therapy approach for C9orf72-linked ALS M Cappella Inaugural Symposium of the Sorbonne Université" Stem Cells and Regenerative …, 2022 | | 2022 |
| An in vitro model to understand the C9orf72-linked Amyotrophic Lateral Sclerosis features in skeletal muscle MT Daher, M Cappella, A Ricupero, CN Van, A Bigot, V Mouly, D Bohl, ... 7th International Congress of Myology, 2022 | | 2022 |
| STUDY OF POTENTIAL OFF-TARGET CANDIDATE SITES FOR ANTISENSE SEQUENCES INDUCING EXON SKIPPING IN SOD1-LINKED AMYOTROPHIC LATERAL SCLEROSIS S Elouej, D Marine, M Cappella, M Cohen-Tannoudji, S Astord, T Marais, ... JR6 FILSLAN-ARSLA, 2020 | | 2020 |
| Development and characterization of in vitro models to test the efficiency of gene therapy approaches in SOD1-linked Amyotrophic lateral sclerosis M Delamare, S Elouej, A Bigot, M Cappella, M Biferi NEUROMUSCULAR DISORDERS 30, S79-S79, 2020 | | 2020 |
| HEREDITARY NEUROPATHIES & ALS: P. 115 Analysis of off-target effects of antisense sequence inducing exon skipping in SOD1-linked amyotrophic lateral sclerosis S Elouej, M Delamare, M Cappella, M Cohen-Tannoudji, S Astord, ... Neuromuscular Disorders 30, S80, 2020 | | 2020 |
| HEREDITARY NEUROPATHIES & ALS: P. 110 Development and characterization of in vitro models to test the efficiency of gene therapy approaches in SOD1-linked Amyotrophic lateral … M Delamare, S Elouej, A Bigot, M Cappella, M Biferi Neuromuscular Disorders 30, S79, 2020 | | 2020 |
| Development and characterization of in vitro models to test the efficiency of gene therapy approaches in SOD1-linked Amyotrophic lateral sclerosis D Marine, S Elouej, A Bigot, M Cappella, MG Biferi WMS, 2020 | | 2020 |
| Analysis of off-target effects of Antisense Sequence inducing Exon Skipping in SOD1-linked Amyotrophic Lateral Sclerosis S Elouej, D Marine, M Cappella, M Cohen-Tannoudji, S Astord, T Marais, ... WMS, 2020 | | 2020 |
| Gene therapy approach for C9orf72-familial Amyotrophic Lateral Sclerosis to induce degradation of RNA containing repeats M Cappella, M Cohen-Tannoudji, A Besse, A Bigot, V Mouly, CL Omar, ... 6th International Congress of Myology, 2019 | | 2019 |
| Gene therapy approaches for familial ALS M Cappella, M Cohen-Tannoudji, T Marais, S Astord, A Besse, B Giroux, ... 29th International Symposium on ALS/MND, 2018 | | 2018 |
| AAV-mediated gene therapy for fALS M Cappella, M Cohen-Tannoudji, T Marais, S Astord, A Besse, B Giroux, ... 26th ESGCT Congress, 2018 | | 2018 |
