Gene Therapy via Trans-Splicing for LMNA-Related Congenital Muscular Dystrophy

We assessed the potential of Lmna-mRNA repair by spliceosome-mediated RNA trans-splicing as a therapeutic approach for LMNA-related congenital muscular dystrophy. This gene therapy strategy leads to reduction of mutated transcript expression for the benefit of corresponding wild-type (WT) transcript...

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Principais autores:	Feriel Azibani, Astrid Brull, Ludovic Arandel, Maud Beuvin, Isabelle Nelson, Arnaud Jollet, Esma Ziat, Bernard Prudhon, Sofia Benkhelifa-Ziyyat, Marc Bitoun, Stéphanie Lorain, Gisèle Bonne, Anne T. Bertrand
格式:	Artigo
語言:	Inglês
出版:	Elsevier 2018-03-01
叢編:	Molecular Therapy: Nucleic Acids
主題:	Lmna lamin A/C LMNA-related congenital muscular dystrophy trans-splicing gene therapy
在線閱讀:	http://www.sciencedirect.com/science/article/pii/S2162253117303116
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