GeneSilencer-V

GeneSilencer-V is an innovative gene therapy platform designed to silence the extra chromosome responsible for trisomy disorders like Down syndrome. By delivering a specially engineered miniXIST RNA via targeted AAV vectors, it selectively inactivates the surplus chromosome without affecting normal chromosomes. This approach offers a potential permanent, precise treatment that addresses the root genetic cause of these conditions, marking a significant advancement beyond current symptom-focused therapies.