Our Mission

The Faculty of the Horae Gene Therapy Center is dedicated to develop therapeutic approaches for rare inherited disease for which there is no cure. We utilize 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.

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, and Huntington's disease. 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.

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