Recent advances in antisense oligonucleotide therapy in genetic neuromuscular diseases
Genetic neuromuscular diseases are caused by defective expression of nuclear or mitochondrial genes. Mutant genes may reduce expression of wild-type proteins, and strategies to activate expression of the wild-type proteins might provide therapeutic benefits. Also, a toxic mutant protein may cause ce...
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| Format: | Article |
| Language: | English |
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Wolters Kluwer Medknow Publications
2018-01-01
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| Series: | Annals of Indian Academy of Neurology |
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| Online Access: | http://www.annalsofian.org/article.asp?issn=0972-2327;year=2018;volume=21;issue=1;spage=3;epage=8;aulast=Verma |