Award
NIH Reporter #11316829
Investigating the Feasibility of Gene Therapy for the Treatment of TBRS
Recipient
Washington University
Awarded
February 16, 2026
Identifier
11316829
Research project awarded to Washington University to investigate gene therapy for TBRS using advanced genetic tools and mouse models, aiming to restore DNMT3A function postnatally.
Description
This project focuses on studying the potential for gene therapy to treat Tatton Brown Rahman Syndrome (TBRS). It involves advanced genetic tools, mouse models, spatial transcriptomics, and gene therapy approaches using adeno-associated viruses. The work aims to assess the feasibility of postnatal DNMT3A restoration to rescue molecular, cellular, and behavioral deficits. The project is conducted at Washington University in Saint Louis, supported by collaborations with experts in neurodevelopmental disorders, genomics, and neurobiology.