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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

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