Title: | Skipping Multiple Exons to Treat DMD-Promises and Challenges |
Journal : | Biomedicines, 6, 1 |
Authors: | Aslesh T, Author ; Maruyama R ; Yokota T |
Material Type: | Article |
Publication Date: | 2018 |
Languages: | English |
Keywords : | animal models ; antisense oligonucleotides ; CRISPR/Cas9 ; drug marketing regulation in France ; Duchenne muscular dystrophy ; DYS gene ; exon skipping ; morpholinos ; therapeutic prospects |
Keywords: | Clustered Regularly Interspaced Short Palindromic Repeat/CRISPR associated protein 9 (CRISPR/Cas9)-mediated genome editing;Duchenne/Becker muscular dystrophy (DMD/BMD);actin binding domain (ABD);antisense oligonucleotides (AOs);canine X-linked muscular dystrophy (CXMD);eteplirsen;golden retriever muscular dystrophy (GRMD);golodirsen;morpholino);multi-exon skipping;phosphorodiamidate morpholino oligomer (PMO); |
Pubmed / DOI : | DOI : 10.3390/biomedicines6010001 / Pubmed : 29301272 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/29301272 |