Saturday, April 20, 2013

New scholarship research into Friedreich Ataxia, heart and eye dysfunction

CERA student receives prestigious Gustav Nossal Scholarship for his research into Friedreich Ataxia .
CERA (Centre for Eye Research Australia).09 April, 2013

Duncan is a PhD student in CERA's Neuroregeneration Research Unit, he uses stem cells generated in the laboratory from FRDA patients' own skin, to grow certain types of cells found in the heart and eye. These cells will be used to better understand the pathology of FRDA, to conduct basic research on the disease and to test new drugs, prior to conducting clinical trials.

Gene Therapy for Rare Diseases: Summary of a National Institutes of Health Workshop, September 13, 2012

Gene Therapy for Rare Diseases: Summary of a National Institutes of Health Workshop, September 13, 2012 . Marina O'Reilly, Donald B. Kohn, Jeffrey Bartlett, Janet Benson, Philip J. Brooks, Barry J. Byrne, Carlos Camozzi, Kenneth Cornetta, Ronald G. Crystal, Yuman Fong, Linda Gargiulo, Rashmi Gopal-Srivastava, Katherine A. High, Samuel G. Jacobson, Robert C. Jambou, Maureen Montgomery, Eugene Rosenthal, R. Jude Samulski, Sonia I. Skarlatos, Brian Sorrentino, James M. Wilson, Yun Xie, and Jacqueline Corrigan-Curay. Human Gene Therapy. April 2013, 24(4): 355-362. doi:10.1089/hum.2013.064.

Gene therapy has shown clinical efficacy for several rare diseases, using different approaches and vectors.