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Antisense oligonucleotide therapies for monogenic disorders.
Med. Genet. 37, 179-187 (2025)
Antisense oligonucleotides (ASOs) are a promising therapeutic modality for monogenic disorders, offering precise RNA-targeting strategies to modulate gene expression. Despite challenges in delivery, toxicity, and off-target effects, ASO therapies have advanced rapidly, with several approved treatments and numerous candidates in clinical development. Their application spans neurogenetic, metabolic, and oncologic disorders, also with emerging n-of-1 approaches for ultra-rare conditions. This review describes the different mechanism of how ASOs work depending on their chemistry and discusses the considerations of which patients could be amendable for treatment highlighting the role of human genetics for decision making.
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Publication type
Article: Journal article
Document type
Scientific Article
Keywords
Antisense Oligonucleotides ; Gene Therapy ; Precision Medicine ; Rna Therapeutics; Hepatotoxicity; Translation
ISSN (print) / ISBN
0936-5931
e-ISSN
1863-5490
Journal
Medizinische Genetik
Quellenangaben
Volume: 37,
Issue: 3,
Pages: 179-187
Publisher
Springer
Publishing Place
Genthiner Strasse 13, D-10785 Berlin, Germany
Non-patent literature
Publications
Reviewing status
Peer reviewed
Institute(s)
Institute of Neurogenomics (ING)