Genome Editing may be the Next Step in Genetic Therapy

In what could prove to be a major breakthrough in genetic therapies, a group of American researchers managed to repair a faulty gene by making the cells to create a new healthy gene in mice. The research was conducted by scientists at the Children's Hospital of Philadelphia who “edited” the gene by injecting the mice with enzymes in order to destroy the faulty gene and force the cells to launch an emergency repair operation.

The enzymes also contained DNA templates for healthy clotting genes and thus instead of merely repairing the faulty gene, the cells created a new healthy gene.

The research was published in the journal Nature and has given hope that new genetic therapies could be developed for humans in the near future.



Source-Medindia