Hematopoietic stem cell gene therapy for the treatment of SYNGAP1-related non-specific intellectual disability.
Joseph S AndersonAlyse L LodigianiCamilla M BarbaduomoJulie R BeeglePublished 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.