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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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Glavni autori: 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
Format: Artigo
Jezik:Inglês
Izdano: Elsevier 2018-03-01
Serija:Molecular Therapy: Nucleic Acids
Teme:
Lmna
lamin A/C
LMNA-related congenital muscular dystrophy
trans-splicing
gene therapy
Online pristup:http://www.sciencedirect.com/science/article/pii/S2162253117303116
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