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 |
| _version_ | 1818857754069041152 |
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| author | Ryan Gotesman |
| author_facet | Ryan Gotesman |
| author_sort | Ryan Gotesman |
| collection | DOAJ |
| description | 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-approved drugs for treating spinal muscular atrophy and Duchenne muscular dystrophy. Recent trials also suggest ASOs may be effective in combating Huntington’s disease, amyotrophic lateral sclerosis and hereditary transthyretin amyloidosis. This article highlights ASOs’ mechanism of action, their use in treating neuromuscular disease and future obstacles the gene therapy must overcome, providing an update on the state of ASO technology. |
| first_indexed | 2024-12-19T08:45:25Z |
| format | Article |
| id | doaj.art-b47b743081164a33b5dd0614365bb66f |
| institution | Directory Open Access Journal |
| issn | 2292-650X 2292-6518 |
| language | English |
| last_indexed | 2024-12-19T08:45:25Z |
| publishDate | 2018-11-01 |
| publisher | University of Ottawa |
| record_format | Article |
| series | University of Ottawa Journal of Medicine |
| spelling | doaj.art-b47b743081164a33b5dd0614365bb66f2022-12-21T20:28:49ZengUniversity of OttawaUniversity of Ottawa Journal of Medicine2292-650X2292-65182018-11-018210.18192/uojm.v8i2.3462Antisense Oligonucleotide Gene Therapy for Neuromuscular DisordersRyan Gotesman0University of Ottawa, Faculty of MedicineAntisense 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-approved drugs for treating spinal muscular atrophy and Duchenne muscular dystrophy. Recent trials also suggest ASOs may be effective in combating Huntington’s disease, amyotrophic lateral sclerosis and hereditary transthyretin amyloidosis. This article highlights ASOs’ mechanism of action, their use in treating neuromuscular disease and future obstacles the gene therapy must overcome, providing an update on the state of ASO technology.https://uottawa.scholarsportal.info/ottawa/index.php/uojm-jmuo/article/view/3462antisense oligonucleotidesASOneuromuscular disordersgene therapy |
| spellingShingle | Ryan Gotesman Antisense Oligonucleotide Gene Therapy for Neuromuscular Disorders University of Ottawa Journal of Medicine antisense oligonucleotides ASO neuromuscular disorders gene therapy |
| title | Antisense Oligonucleotide Gene Therapy for Neuromuscular Disorders |
| title_full | Antisense Oligonucleotide Gene Therapy for Neuromuscular Disorders |
| title_fullStr | Antisense Oligonucleotide Gene Therapy for Neuromuscular Disorders |
| title_full_unstemmed | Antisense Oligonucleotide Gene Therapy for Neuromuscular Disorders |
| title_short | Antisense Oligonucleotide Gene Therapy for Neuromuscular Disorders |
| title_sort | antisense oligonucleotide gene therapy for neuromuscular disorders |
| topic | antisense oligonucleotides ASO neuromuscular disorders gene therapy |
| url | https://uottawa.scholarsportal.info/ottawa/index.php/uojm-jmuo/article/view/3462 |
| work_keys_str_mv | AT ryangotesman antisenseoligonucleotidegenetherapyforneuromusculardisorders |