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...

Full description

Saved in:
Bibliographic Details
Published inNervenarzt Vol. 91; no. 4; pp. 318 - 323
Main Authors Lehmann Urban, Diana, Schneider, Ilka
Format Journal Article
LanguageGerman
Published Germany 01.04.2020
Subjects
Cell-based treatment
Antisense oligonucleotide
Gene therapy
Myotonic dystrophy 1
Muscular dystrophy, Duchenne
Online AccessGet full text

Cover

More Information
Summary: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.
Bibliography:ObjectType-Article-2
SourceType-Scholarly Journals-1
ObjectType-Feature-3
content type line 23
ObjectType-Review-1
ISSN:1433-0407
DOI:10.1007/s00115-020-00870-8