Clean correction of a patient’s genetic mutation: New gene therapy methods accurately correct mutation in patient’s stem cells

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 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.