Welcome to our Gene Therapy Center!
Gene Therapy Promise
Treating human diseases by utilizing gene therapy strategies have taken the scientific world by storm. Improved delivery tools (i.e. AAVs) and novel therapeutic strategies are proving that gene therapy has the promise of successfully threating a wide spectrum of diseases that were once uncurable. Read more about what is gene therapy, what are the tools and current strategies scientists use to advance the field and cure disease.
The Faculty of the Horae Gene Therapy Center is dedicated to develop therapeutic approaches for rare inherited diseases for which there is no cure utilizing state of the art technologies to correct the genetic mutations. Our focus is on AAT Deficiency, Amyotrophic Lateral Sclerosis (Lou Genrig's Disease), Canavan Disease, Cystic Fibrosis, Tay-Sachs & Sandhoff diseases, Retinitis Pigmentosa, Huntington's disease, Hypercholesterimia and Cardiac arrhythmia.
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Under the umbrella of the ATC, UMass Medical School formed the Gene Therapy Center (GTC) emphasizing the promise that lies within the application of the recombinant adeno-associated virus; RNA Therapeutics Institute (RTI) featuring novel strategies for using the RNAi mechanism to silence the action of individual genes and The Center for Stem Cell Biology and Regenerative Medicine seeking to unlock the enormous promise to elucidate disease mechanisms inherent in humans.
The Horae Gene Therapy Center is always interested in possible partnerships with both indivituals and organizations. Some possible partnerships include knowledge exchange, consulting, collaborations with academia and/or industry, reagent exchange, sponsored research, and spinout activities. We encourage you to contact us and learn more how we can partner together to advance the area of gene therapy.
We believe that great scientific discoveries happen when people collaborate.
Let's have the next big breakthrough together!