Development of AAV vectors for the treatment of glycogen storage disease type Ib

  • Proprietary hG6PT vector displays ubiquitous tissue expression that can be used to enhance gene expression for a variety of gene therapy applications
  • Human-optimized AAV mediated vector gene therapy for the treatment of glycogen storage disease type Ib
  • Treatment with recombined AAV vector showed significantly improved outcomes in GSD-Ib mice

Website

https://uconn.flintbox.com/technologies/F3C48290CCC74218857DB8E9DF9C4609

Potential Applications

  • Recombinant AAV vector and DNA can be used as a gene therapy treatment for glycogen storage disease type Ib
  • Platform technology can be used in a variety of gene therapy applications

Contact Information

Name: Amit Kumar

Email: a.kumar@uconn.edu

Phone: (860) 486-6938