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

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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Main Authors: Feriel Azibani (Author), Astrid Brull (Author), Ludovic Arandel (Author), Maud Beuvin (Author), Isabelle Nelson (Author), Arnaud Jollet (Author), Esma Ziat (Author), Bernard Prudhon (Author), Sofia Benkhelifa-Ziyyat (Author), Marc Bitoun (Author), Stéphanie Lorain (Author), Gisèle Bonne (Author), Anne T. Bertrand (Author)
Format: Book
Published: Elsevier, 2018-03-01T00:00:00Z.
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