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Gene Therapy Improves Vision in Patients with Congenital Retinal Disease
Date:4/27/2008

, cure and prevention of human genetic diseases. TIGEM's mission is to understand the mechanisms of genetic diseases and to develop preventive and therapeutic strategies. Since its establishment, the Institute has grown considerably. It now consists of a large fully refurbished site, and comprises 13 independent research groups with over 170 members including graduate students, postdoctorate fellows, technicians and administration. The scope of the science currently covered at TIGEM spans three disease research area: developmental disorders, inborn errors of metabolism and inherited eye diseases. Research approaches include cell biology, functional genomics, systems biology and gene therapy. TIGEM offers training programs in medical and human genetics, in cooperation with local and international universities such as the British Open University. Research activity at TIGEM is supported by core facilities dedicated to providing state-of-the-art technology as well as housekeeping assistance.

About the Howard Hughes Medical Institute (HHMI): HHMI, a non-profit medical research organization that ranks as one of the nation's largest philanthropies, plays a powerful role in advancing biomedical research and science education in the U.S. In the past two decades HHMI has made investments of more than $8.3 billion for the support, training, and education of the nation's most creative and promising scientists. HHMI's flagship program in biomedical research rests on the conviction that scientists of exceptional talent, commitment, and imagination will make fundamental biological discoveries for the betterment of human health if they receive the resources, time, and freedom to pursue challenging questions. The 298 investigators of HHMI, selected through rigorous national competitions, include 12 Nobel Prize Winners and 122 members of the National Academy of Sciences. Founded in 1953 by Howard R. Hughes, the aviator and industrialist, HHMI is headquartered in Chevy Chase,
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SOURCE The Children's Hospital of Philadelphia; University ofPennsylvania
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