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Khabre desk
Recent reporting, analysis and explainers from the antisense oligonucleotides desk.
health
A man with slowly progressing ALS caused by a rare CHCHD10 mutation showed improved motor-function scores and a biomarker associated with nerve damage returned to the normal reference range one year after receiving a mutation-targeted antisense oligonucleotide treatment.
health
A medRxiv preprint maps a COL4A5 intronic mutation hotspot and reports that one antisense oligonucleotide restored normal RNA splicing and collagen expression for multiple variants in patient-derived cells. The work is a preclinical study, with no treatment of patients reported.