Stanford University researchers restore cellular function in Timothy syndrome organoids using antisense oligonucleotides (ASOs), a gene-based therapy, targeting the mutated CACNA1C gene.

Stanford University researchers restore cellular function in Timothy syndrome organoids using antisense oligonucleotides (ASOs), a gene-based therapy. The study, funded by the National Institutes of Health, targets the mutated CACNA1C gene and offers potential for new treatments, providing broader insights into rare genetic conditions and mental disorders.