Welcome to our Gene Therapy Center!
We are excited to announce that Dr. Jennifer Adair will be joining UMass Chan Medical School as a Professor and Associate Director of the Horae Gene Therapy Center (HGTC)! Dr. Adair is a world-leading scientist whose research focuses on isolating stem and immune cell populations, genetically modifying them with cutting-edge CRISPR technology, and then delivering them to patients as a "living drug" as well as using nanoparticles as a platform to deliver gene therapy to patient cells. She is an internationally recognized expert in using these approaches to develop novel treatments for genetic, malignant, and infectious diseases as well as in the advancement of gene therapy vector technologies and manufacturing. In addition to her work in the scientific community, Dr. Adair is a leading advocate to improve global healthcare equity and improve access to medicines for patients in developing countries. Along with Dr. Cissy Kityo-Mutuluuza, Dr. Adair established the Global Gene Therapy Initiative working group to overcome barriers to genetic medicine being available to patients in low- and middle-income countries, such as limited resources, lack of infrastructure, and regulations governing international transfer of technologies. Dr. Adair has pioneered an innovative technology platform called “gene therapy in a box” as a portable and low-cost system for manufacturing living drugs to make therapies more globally accessible. In its 15-year history, the HGTC at UMass Chan has been a leading institution in the development of recombinant adeno-associated virus-based in vivo gene therapy approaches to treat human disease. As the Associate Director, Dr. Adair will oversee the establishment of an Ex Vivo Gene Therapy and Editing Core and lead the development of lentiviral and nonviral platforms in the HGTC to complement the established expertise of using in vivo approaches. Dr. Adair’s exceptional scientific expertise and leadership will help guide and grow the HGTC as a world-leading center for both in vivo and ex vivo approaches to create new genetic medicines for patients with inherited and acquired diseases. |
Gene Therapy Promise
Explore what is gene therapy, current strategies and delivery tools
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.
Breakthrough Research
Developing breakthrough gene therapies for rare genetic disorders
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.
GTC Buzz: What's Hot!
Read the latest news covering our research and discoveries
Supportive Ecosystem
We are part of the Advanced Therapeutics Cluster (ATC)
Under the umbrella of the ATC, UMass Chan 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.
Partner Opportunities
Learn more how we can partner together
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!