Antisense Oligonucleotide Gene Therapy for Neuromuscular Disorders
Antisense oligonucleotides (ASOs) are synthetic, single-stranded DNA molecules that can bind to specific mRNA sequences and alter protein expression. ASO gene therapies are leading to breakthroughs in the treatment of once intractable neuromuscular disorders. In 2016, ASOs became the first FDA-appro...
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| Format: | Article |
| Language: | English |
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University of Ottawa
2018-11-01
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| Series: | University of Ottawa Journal of Medicine |
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| Online Access: | https://uottawa.scholarsportal.info/ottawa/index.php/uojm-jmuo/article/view/3462 |