oligonucleotide ASO investigational
Selected indexed studies
- Potential disease-modifying therapies for Huntington's disease: lessons learned and future opportunities. (Lancet Neurol, 2022) [PMID:35716694]
- AAV-mediated exon skipping therapy for Usher syndrome, type 2A. (Mol Ther, 2026) [PMID:40994011]
- An ASO therapy for Angelman syndrome that targets an evolutionarily conserved region at the start of the UBE3A-AS transcript. (Sci Transl Med, 2023) [PMID:36947593]
_Worker-drafted node — pending editorial review._
Connections
No connections recorded yet.
Sources
- Lp(a)-Lowering Agents in Development: A New Era in Tackling the Burden of Cardiovascular Risk? (2025) pubmed
- Safety and efficacy of therapies for chylomicronemia. (2022) pubmed
- Potential disease-modifying therapies for Huntington's disease: lessons learned and future opportunities. (2022) pubmed
- AAV-mediated exon skipping therapy for Usher syndrome, type 2A. (2026) pubmed
- An ASO therapy for Angelman syndrome that targets an evolutionarily conserved region at the start of the UBE3A-AS transcript. (2023) pubmed
- Current Clinical Trials for Treating Elevated Lipoprotein(a). (2025) pubmed
- Investigational RNA Interference Agents for Hepatitis B. (2025) pubmed
- Emerging, novel gene-modulating therapies for transthyretin amyloid cardiomyopathy. (2025) pubmed
- Brain somatic mutations as RNA therapeutic targets in neurological disorders. (2022) pubmed
- Preclinical study and parallel phase II trial evaluating antisense STAT3 oligonucleotide and checkpoint blockade for advanced pancreatic, non-small cell lung cancer and mismatch repair-deficient colorectal cancer. (2024) pubmed