Targeting RNA opens therapeutic avenues for Timothy syndrome

Nature. 2024 Apr;628(8009):730-732. doi: 10.1038/d41586-024-00911-1.
No abstract available

Keywords: Autism spectrum disorders; Gene therapy; Stem cells; Therapeutics.

Publication types

  • News

MeSH terms

  • Animals
  • Autistic Disorder* / drug therapy
  • Autistic Disorder* / genetics
  • Autistic Disorder* / therapy
  • Humans
  • Long QT Syndrome* / drug therapy
  • Long QT Syndrome* / genetics
  • Mice
  • Molecular Targeted Therapy
  • Syndactyly*

Supplementary concepts

  • Timothy syndrome