M
Marisa Cappella
Researcher at University of Paris
Publications - 7
Citations - 204
Marisa Cappella is an academic researcher from University of Paris. The author has contributed to research in topics: microRNA & Alternative splicing. The author has an hindex of 5, co-authored 7 publications receiving 146 citations. Previous affiliations of Marisa Cappella include Sapienza University of Rome & National Research Council.
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Journal ArticleDOI
Gene Therapy for ALS-A Perspective.
TL;DR: Gene therapy approaches, involving the delivery of antisense oligonucleotides into the central nervous system (CNS) are being tested in clinical trials for patients with mutations in SOD1 or C9orf72 genes, and are potentially a promising avenue to develop an efficient and safe cure for ALS.
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CRISPR/Cas9-Mediated Deletion of CTG Expansions Recovers Normal Phenotype in Myogenic Cells Derived from Myotonic Dystrophy 1 Patients
Claudia Provenzano,Marisa Cappella,Rea Valaperta,Rosanna Cardani,Giovanni Meola,Fabio Martelli,Beatrice Cardinali,Germana Falcone +7 more
TL;DR: Using the CRISPR/Cas9 gene-editing system, the repeat expansions were removed, therefore preventing nuclear foci formation and splicing alterations in myotonic dystrophy cells, and affected cells can be permanently reverted to a normal phenotype.
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MicroRNA-222 regulates muscle alternative splicing through Rbm24 during differentiation of skeletal muscle cells.
Beatrice Cardinali,Marisa Cappella,Marisa Cappella,Claudia Provenzano,Jose Manuel Garcia-Manteiga,Dejan Lazarevic,Davide Cittaro,Fabio Martelli,Germana Falcone +8 more
TL;DR: A new function of microRNA-222 leading to alteration of myogenic differentiation at the level of alternative splicing is identified, and evidence that this effect is mediated by Rbm24 protein is provided.
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Beyond the Traditional Clinical Trials for Amyotrophic Lateral Sclerosis and The Future Impact of Gene Therapy.
TL;DR: Clinical use of antisense oligonucleotides in three familial forms of ALS, caused by mutations in SOD1, C9orf72 and FUS genes are described, highlighting the potential of gene therapy as a transforming technology that will have a huge impact on treatment perspective for ALS patients and on the design of future clinical trials.
Journal ArticleDOI
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.
Marisa Cappella,Alessandra Perfetti,Beatrice Cardinali,Jose Manuel Garcia-Manteiga,Matteo Carrara,Claudia Provenzano,Paola Fuschi,Rosanna Cardani,Laura Valentina Renna,Giovanni Meola,Germana Falcone,Fabio Martelli +11 more
TL;DR: Functional relevant miRNA/mRNA interactions were identified in skeletal muscles of DM1 patients, highlighting the dysfunction of miR-29c and ASB2.