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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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Publikationstyp
Artikel: Journalartikel
Dokumenttyp
Wissenschaftlicher Artikel
Schlagwörter
Antisense Oligonucleotides ; Gene Therapy ; Precision Medicine ; Rna Therapeutics; Hepatotoxicity; Translation
ISSN (print) / ISBN
0936-5931
e-ISSN
1863-5490
Zeitschrift
Medizinische Genetik
Quellenangaben
Band: 37,
Heft: 3,
Seiten: 179-187
Verlag
Springer
Verlagsort
Genthiner Strasse 13, D-10785 Berlin, Germany
Nichtpatentliteratur
Publikationen
Begutachtungsstatus
Peer reviewed
Institut(e)
Institute of Neurogenomics (ING)