Antisense Morpholino-Based In Vitro Correction of a Pseudoexon-Generating Variant in the SGCB Gene

Limb-girdle muscular dystrophies (LGMD) are clinically and genetically heterogenous presentations displaying predominantly proximal muscle weakness due to the loss of skeletal muscle fibers. Beta-sarcoglycanopathy (LGMDR4) results from biallelic molecular defects in SGCB and features pediatric onset...

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Detalles Bibliográficos
Autores principales: Magri, Francesca, Zanotti, Simona, Salani, Sabrina, Fortunato, Francesco, Ciscato, Patrizia, Gerevini, Simonetta, Maggi, Lorenzo, Sciacco, Monica, Moggio, Maurizio, Corti, Stefania, Bresolin, Nereo, Comi, Giacomo Pietro, Ronchi, Dario
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9456520/
https://www.ncbi.nlm.nih.gov/pubmed/36077211
http://dx.doi.org/10.3390/ijms23179817

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9456520/
https://www.ncbi.nlm.nih.gov/pubmed/36077211
http://dx.doi.org/10.3390/ijms23179817

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