New Approach for Antisense Oligonucleotide-Mediated Exon Skipping in Duchenne Muscular Dystrophy.
Duchenne Muscular Dystrophy (DMD) is a lethalmuscle disorder characterized by mutations in the DMD gene. These mutations primarily disrupt the reading frame, resulting in the absence of functional dystrophin protein. Exon skipping, which involves the use of antisense oligonucleotides is a promising...
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| Format: | Journal article |
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2012
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| _version_ | 1797056984695963648 |
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| author | Aoki, Y Nagata, T Takeda, S |
| author_facet | Aoki, Y Nagata, T Takeda, S |
| author_sort | Aoki, Y |
| collection | OXFORD |
| description | Duchenne Muscular Dystrophy (DMD) is a lethalmuscle disorder characterized by mutations in the DMD gene. These mutations primarily disrupt the reading frame, resulting in the absence of functional dystrophin protein. Exon skipping, which involves the use of antisense oligonucleotides is a promising therapeutic approach for DMD, and clinical trials on exon skipping are currently underway in DMD patients. Recently, stable and less-toxic antisense oligonucleotides with higher efficacy have been developed in mouse and dog models of DMD. This review highlights a new approach for antisense oligonucleotide-based therapeutics for DMD, particularly for exon skipping-based methods. |
| first_indexed | 2024-03-06T19:30:05Z |
| format | Journal article |
| id | oxford-uuid:1d27c024-d997-45b3-a3d1-f4688afa3238 |
| institution | University of Oxford |
| last_indexed | 2024-03-06T19:30:05Z |
| publishDate | 2012 |
| record_format | dspace |
| spelling | oxford-uuid:1d27c024-d997-45b3-a3d1-f4688afa32382022-03-26T11:09:21ZNew Approach for Antisense Oligonucleotide-Mediated Exon Skipping in Duchenne Muscular Dystrophy.Journal articlehttp://purl.org/coar/resource_type/c_dcae04bcuuid:1d27c024-d997-45b3-a3d1-f4688afa3238Symplectic Elements at Oxford2012Aoki, YNagata, TTakeda, SDuchenne Muscular Dystrophy (DMD) is a lethalmuscle disorder characterized by mutations in the DMD gene. These mutations primarily disrupt the reading frame, resulting in the absence of functional dystrophin protein. Exon skipping, which involves the use of antisense oligonucleotides is a promising therapeutic approach for DMD, and clinical trials on exon skipping are currently underway in DMD patients. Recently, stable and less-toxic antisense oligonucleotides with higher efficacy have been developed in mouse and dog models of DMD. This review highlights a new approach for antisense oligonucleotide-based therapeutics for DMD, particularly for exon skipping-based methods. |
| spellingShingle | Aoki, Y Nagata, T Takeda, S New Approach for Antisense Oligonucleotide-Mediated Exon Skipping in Duchenne Muscular Dystrophy. |
| title | New Approach for Antisense Oligonucleotide-Mediated Exon Skipping in Duchenne Muscular Dystrophy. |
| title_full | New Approach for Antisense Oligonucleotide-Mediated Exon Skipping in Duchenne Muscular Dystrophy. |
| title_fullStr | New Approach for Antisense Oligonucleotide-Mediated Exon Skipping in Duchenne Muscular Dystrophy. |
| title_full_unstemmed | New Approach for Antisense Oligonucleotide-Mediated Exon Skipping in Duchenne Muscular Dystrophy. |
| title_short | New Approach for Antisense Oligonucleotide-Mediated Exon Skipping in Duchenne Muscular Dystrophy. |
| title_sort | new approach for antisense oligonucleotide mediated exon skipping in duchenne muscular dystrophy |
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