Progress in the Treatment of Antisense Oligonucleotides in Duchenne Muscular Dystrophy
In recent years, antisense oligonucleotide (ASO) has been very active in the field of rare disease research and development, especially in Duchenne muscular dystrophy, where it made a major breakthrough. Duchenne muscular dystrophy (DMD) is a rare childhood myopathy caused by mutations in the dystro...
| Main Authors: | , , , , , , |
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| Format: | Article |
| Language: | zho |
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Editorial Office of Journal of Rare Diseases
2022-04-01
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| Series: | 罕见病研究 |
| Subjects: | |
| Online Access: | https://jrd.chard.org.cn/article/doi/10.12376/j.issn.2097-0501.2022.02.015 |
| _version_ | 1797369806585856000 |
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| author | XU Tingting ZUO Wei LIU Xin FAN Qianqian GAO Yang LIANG Chunsu ZHANG Bo |
| author_facet | XU Tingting ZUO Wei LIU Xin FAN Qianqian GAO Yang LIANG Chunsu ZHANG Bo |
| author_sort | XU Tingting |
| collection | DOAJ |
| description | In recent years, antisense oligonucleotide (ASO) has been very active in the field of rare disease research and development, especially in Duchenne muscular dystrophy, where it made a major breakthrough. Duchenne muscular dystrophy (DMD) is a rare childhood myopathy caused by mutations in the dystrophin gene. Currently, the four ASO drugs approved internationally for DMD are all targeted at dystrophin, including eteplirsen, golodirsen, viltolarsen and casimersen. They all belong to phosphorodiamidate morpholino oligomers (PMO) antisense oligonucleotide drugs, so that their pharmacokinetic characteristics are similar. The drugs quickly spread to other tissues after intravenous administration. Because of the electrical neutrality of the PMO, they have a low binding rate to plasma proteins and are quickly metabolized by the kidney and excreted in the urine as archetypes. In addition, the likelihood of drug-drug interactions of ASO is low. Existing clinical studies have shown that they have certain clinical benefits and good tolerability, bringing new options for DMD treatment. This paper mainly discusses the pharmacological effects, pharmacokinetic characteristics, efficacy, and safety of ASO drugs for the treatment of DMD, hoping to provide scientific reference for the rational and safe clinical use of such drugs. |
| first_indexed | 2024-03-08T17:52:14Z |
| format | Article |
| id | doaj.art-7e9f9ad1b9754ee89ec6384f6c93c122 |
| institution | Directory Open Access Journal |
| issn | 2097-0501 |
| language | zho |
| last_indexed | 2024-03-08T17:52:14Z |
| publishDate | 2022-04-01 |
| publisher | Editorial Office of Journal of Rare Diseases |
| record_format | Article |
| series | 罕见病研究 |
| spelling | doaj.art-7e9f9ad1b9754ee89ec6384f6c93c1222024-01-02T07:14:47ZzhoEditorial Office of Journal of Rare Diseases罕见病研究2097-05012022-04-011219920510.12376/j.issn.2097-0501.2022.02.015Progress in the Treatment of Antisense Oligonucleotides in Duchenne Muscular DystrophyXU TingtingZUO WeiLIU XinFAN QianqianGAO YangLIANG ChunsuZHANG BoIn recent years, antisense oligonucleotide (ASO) has been very active in the field of rare disease research and development, especially in Duchenne muscular dystrophy, where it made a major breakthrough. Duchenne muscular dystrophy (DMD) is a rare childhood myopathy caused by mutations in the dystrophin gene. Currently, the four ASO drugs approved internationally for DMD are all targeted at dystrophin, including eteplirsen, golodirsen, viltolarsen and casimersen. They all belong to phosphorodiamidate morpholino oligomers (PMO) antisense oligonucleotide drugs, so that their pharmacokinetic characteristics are similar. The drugs quickly spread to other tissues after intravenous administration. Because of the electrical neutrality of the PMO, they have a low binding rate to plasma proteins and are quickly metabolized by the kidney and excreted in the urine as archetypes. In addition, the likelihood of drug-drug interactions of ASO is low. Existing clinical studies have shown that they have certain clinical benefits and good tolerability, bringing new options for DMD treatment. This paper mainly discusses the pharmacological effects, pharmacokinetic characteristics, efficacy, and safety of ASO drugs for the treatment of DMD, hoping to provide scientific reference for the rational and safe clinical use of such drugs.https://jrd.chard.org.cn/article/doi/10.12376/j.issn.2097-0501.2022.02.015antisense oligonucleotidesduchenne muscular dystrophyrare diseasespharmacokineticsefficacy and safety |
| spellingShingle | XU Tingting ZUO Wei LIU Xin FAN Qianqian GAO Yang LIANG Chunsu ZHANG Bo Progress in the Treatment of Antisense Oligonucleotides in Duchenne Muscular Dystrophy 罕见病研究 antisense oligonucleotides duchenne muscular dystrophy rare diseases pharmacokinetics efficacy and safety |
| title | Progress in the Treatment of Antisense Oligonucleotides in Duchenne Muscular Dystrophy |
| title_full | Progress in the Treatment of Antisense Oligonucleotides in Duchenne Muscular Dystrophy |
| title_fullStr | Progress in the Treatment of Antisense Oligonucleotides in Duchenne Muscular Dystrophy |
| title_full_unstemmed | Progress in the Treatment of Antisense Oligonucleotides in Duchenne Muscular Dystrophy |
| title_short | Progress in the Treatment of Antisense Oligonucleotides in Duchenne Muscular Dystrophy |
| title_sort | progress in the treatment of antisense oligonucleotides in duchenne muscular dystrophy |
| topic | antisense oligonucleotides duchenne muscular dystrophy rare diseases pharmacokinetics efficacy and safety |
| url | https://jrd.chard.org.cn/article/doi/10.12376/j.issn.2097-0501.2022.02.015 |
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