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Gene Therapy - Professor David Lomas

For the first time, scientists have cleanly corrected a gene mutation in a patient’s stem cells, bringing the possibility of patient-specific therapies closer to reality. The team, led by researchers from the Wellcome Trust Sanger Institute and the University of Cambridge, targeted a mutation responsible for cirrhotic liver disease and lung emphysema. Using cutting-edge methods, they corrected the sequence of a patient’s genome, removed all exogenous DNA and showed that the gene worked normally.

The work was co-led by Professor David Lomas, Fellow of St John's College, Cambridge.