Delivering a Therapeutic Gene         Silencing a Disease Gene  

Gene Therapy

Research efforts on the genetic mapping of inherited blinding diseases combined with the Human Genome Project have produced a wealth of data in recent years. Over 100 retina disease genes have now been identified, enabling the research community to consider treatment strategies for hereditary blindness in the post-genomic era. Once a gene mutation has been identified as the cause of blindness in a family, a targeted approach can be designed to attempt to prevent disease progression or restore vision. In addition to developing pharmaceutical, cell transplantation and stem cell strategies, a more direct "genetic medicine" can be considered. The availability of numerous models which mimic human inherited blinding diseases has led to a better understanding of the disease and facilitated the development of potential therapies aimed at elevating a specific genetic disorder. The concept of genetic therapies is simple, to transfer therapeutic genetic material to the eye in order to correct or compensate for the faulty gene.

IoO researchers working in this area:

Professor Robin Ali
Dr James Bainbridge
Professor Shomi Bhattacharya
Mr Frank Larkin
Professor Tony Moore
Dr Andrew Webster
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