Home » Academic life

Gene Therapy - Professor David Lomas

Immunofluorescence image
Immunofluorescence showing the absence of polymeric A1AT protein in hepatocyte-like cells generated from correctediPSCs. All forms of A1AT (left panels) and misfolded polymeric A1AT (middlepanels) are shown.

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.