Antisense-mediated exon skipping: Taking advantage of a trick from Mother Nature to treat rare genetic diseases

Author:

Veltrop Marcel,Aartsma-Rus Annemieke

Publisher

Elsevier BV

Subject

Cell Biology

Reference46 articles.

1. New insights in gene-derived therapy: the example of Duchenne muscular dystrophy;Aartsma-Rus;Ann. N. Y. Acad. Sci,2010

2. Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations;Aartsma-Rus;Hum. Mutat.,2009

3. Lack of myostatin results in excessive muscle growth but impaired force generation;Amthor;Proc. Nat. Acad. Sci. U.S.A,2007

4. Development of multiexon skipping antisense oligonucleotide therapy for Duchenne muscular dystrophy;Aoki;Biomed. Res. Int,2013

5. Pip6-PMO, a new generation of peptide-oligonucleotide conjugates with improved cardiac exon skipping activity for DMD treatment;Betts;Mol. Ther. Nucleic Acids,2012

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