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Antisense Oligonucleotide Therapeutic Approach for Timothy Syndrome

The study focuses on Timothy syndrome (TS), a severe disorder characterized by various neuropsychiatric conditions. TS type 1 (TS1) is linked to a genetic variant in the CACNA1C gene’s exon 8A. Previous research revealed several neuronal phenotypes associated with TS1, including abnormal calcium signaling and impaired neuronal migration. The study aimed to develop a potential therapeutic strategy by targeting exon 8A using antisense oligonucleotides (ASOs). They successfully decreased exon 8A inclusion both in vitro and in vivo, leading to significant improvements in patient-derived cortical organoids and forebrain assembloids. Transplantation of ASO-treated cells also rescued calcium changes and dendrite retraction in patient neurons. This demonstrates the potential of suppressing CACNA1C exon 8A expression as a treatment for TS1, highlighting the effectiveness of a multilevel stem cell-based approach in identifying therapeutic strategies for neurological disorders.

Keywords: Anti-sense oligonucelotide, therapeutics, Timothy Syndrome, neurological disorders

Important Details

  • Validity: Until September 30th, 23:59 German time.
  • Applicability: One experiment per indication (Oncology, Skin & Cosmetics, Organoids).
  • Flexibility: Use the coupon unlimited times until the event ends.
  • Conditions: Applies when proceeding with a contract.

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