Efficient bypass of mutations in dysferlin deficient patient cells by antisense-induced exon skipping-Reference-Cited by-同舟云学术

Efficient bypass of mutations in dysferlin deficient patient cells by antisense-induced exon skipping

Published:2010-02 Issue:2 Volume:31 Page:136-142
ISSN:1059-7794
Container-title:Human Mutation
language:en
Short-container-title:Hum. Mutat.

Author:

Wein Nicolas,Avril Aurélie,Bartoli Marc,Beley Cyriaque,Chaouch Soraya,Laforêt Pascal,Behin Anthony,Butler-Browne Gillian,Mouly Vincent,Krahn Martin,Garcia Luis,Lévy Nicolas

Publisher

Wiley

Subject

Genetics (clinical),Genetics

Link

http://onlinelibrary.wiley.com/wol1/doi/10.1002/humu.21160/fullpdf

Reference28 articles.

1. Antisense-induced multiexon skipping for Duchenne muscular dystrophy makes more sense;Aartsma-Rus;Am J Hum Genet,2004

2. Defective membrane repair in dysferlin-deficient muscular dystrophy;Bansal;Nature,2003

3. Efficient control of gene expression in the hematopoietic system using a single Tet-on inducible lentiviral vector;Barde;Mol Ther,2006

5. Restoration of human dystrophin following transplantation of exon-skipping-engineered DMD patient stem cells into dystrophic mice;Benchaouir;Cell Stem Cell,2007

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