Gene-specific treatment approaches in muscle diseases
Gene-specific treatment for hereditary muscle diseases has made great progress in recent years. The pathomechanisms of many of these diseases could be decrypted using molecular genetic techniques, paving the way for disease-modifying treatment options. A milestone was undoubtedly the successful tran...
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| Published in | Nervenarzt Vol. 91; no. 4; pp. 318 - 323 |
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| Main Authors | , |
| Format | Journal Article |
| Language | German |
| Published |
Germany
01.04.2020
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| Subjects | |
| Online Access | Get full text |
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| Abstract | Gene-specific treatment for hereditary muscle diseases has made great progress in recent years. The pathomechanisms of many of these diseases could be decrypted using molecular genetic techniques, paving the way for disease-modifying treatment options. A milestone was undoubtedly the successful translation of the antisense oligonucleotide (ASO) technology into clinical practice, with gene-specific ASOs being approved for the first time in 2016 for the treatment of spinal muscular atrophy and Duchenne muscular dystrophy. This article reviews recent developments in the field of antisense and gene therapies for hereditary muscle diseases. |
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| AbstractList | Gene-specific treatment for hereditary muscle diseases has made great progress in recent years. The pathomechanisms of many of these diseases could be decrypted using molecular genetic techniques, paving the way for disease-modifying treatment options. A milestone was undoubtedly the successful translation of the antisense oligonucleotide (ASO) technology into clinical practice, with gene-specific ASOs being approved for the first time in 2016 for the treatment of spinal muscular atrophy and Duchenne muscular dystrophy. This article reviews recent developments in the field of antisense and gene therapies for hereditary muscle diseases. |
| Author | Lehmann Urban, Diana Schneider, Ilka |
| Author_xml | – sequence: 1 givenname: Diana surname: Lehmann Urban fullname: Lehmann Urban, Diana email: diana.lehmann@rku.de organization: Neurologische Universitätsklinik, Universitäts- und Rehabilitationskliniken Ulm, Oberer Eselsberg 45, 89081, Ulm, Deutschland. diana.lehmann@rku.de – sequence: 2 givenname: Ilka surname: Schneider fullname: Schneider, Ilka organization: Neurologie Klinikum St. Georg, Leipzig, Deutschland |
| BackLink | https://www.ncbi.nlm.nih.gov/pubmed/32076754$$D View this record in MEDLINE/PubMed |
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| DocumentTitleAlternate | Genspezifische Therapieansätze bei Muskelerkrankungen |
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| Keywords | Cell-based treatment Antisense oligonucleotide Gene therapy Myotonic dystrophy 1 Muscular dystrophy, Duchenne |
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