The Khanna Lab is part of the Horae Gene Therapy Center (GTC), which is dedicated to developing therapeutic approaches for rare inherited disease for which there is no cure such as Canavan Disease, Tay-Sachs and Sandhoff diseases, Retinitis Pigmentosa, Cystic fibrosis, Lou Gehrig's disease, Huntington's disease, as well as therapies for the more common diseases such as Hypercholesterimia and Cardiac arrhythmia. The Center utilizes state of the art technologies to either edit mutated genes that produce disease-causing proteins or introduce a healthy copy of a gene if the mutation results in a non-functional protein.
Horae Gene Therapy holds the promise to treat rare genetic disorders with precision medicine alleviating the suffering of those that are hit by misfortune by a personalized medicine approach. The application of Gene Therapy is also spreading beyond correcting individual gene mutations. Treatments are being developed for common age related diseases such as high cholesterol in the hope that a one time therapy could avoid the daily intake of regular medication reducing the overall burden on patients and the health care system.
The Horae Gene Therapy Center faculty is interdisciplinary covering a wide range of departments such as Pediatrics, Microbiology, Physiology, Biochemistry, Molecular Pharmacology, Neurology, Medicine and Ophthalmology. Medical Doctors and Ph.Ds.' work together to address the medical needs of rare diseases such as Canavan Disease, Tay-Sachs and Sandhoff diseases, Retinitis Pigmentosa, Cystic fibrosis, Lou Gehrig's disease, Huntington's disease and ALS. More common diseases such as cardiac arrhythmia and hypercholesteolemia are also investigated. The hope is to treat a wide spectrum of diseases by various gene therapeutic approaches. Additionally, the University of Massachusetts conducts clinical trial on site and some of these trials are conducted by the investigators at the Gene Therapy center.