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Hematopoietic stem cell gene therapy for the treatment of SYNGAP1-related non-specific intellectual disability.

Joseph S AndersonAlyse L LodigianiCamilla M BarbaduomoJulie R Beegle
Published in: The journal of gene medicine (2024)
These early-stage results highlight the potential of this stem cell gene therapy approach as a treatment strategy for SYNGAP1.
Keyphrases
  • gene therapy
  • intellectual disability
  • early stage
  • stem cells
  • hematopoietic stem cell
  • autism spectrum disorder
  • squamous cell carcinoma
  • bone marrow
  • replacement therapy
  • rectal cancer
  • cell therapy