Genome editing may enable researchers and clinicians to edit genes within the human genome to treat conditions like sickle cell anemia or cystic fibrosis, but the idea for such editing came to Jennifer Doudna at the University of California, Berkeley, as she was studying how bacteria avoid viral infections, NPR reports.
To stave off infection, bacteria slice the viral DNA and make a change to the DNA at that site to disable the virus. If those where and how changes occur could be directed, then any DNA sequence could be modified to how scientists want.
"I thought, wow, if this could work in animal or plant cells, this could be a very, very useful and very powerful tool," Doudna tells NPR.
And CRISPR/Cas9 editing has shown some exciting promise as a faster and cheaper way to make changes to DNA within cells, and could have significance for medicine, NPR notes.
For instance, if two carriers for the cystic fibrosis gene want to avoid passing it on to their child, the CRISPR/Cas9 approach could be employed to change the affected gene. Blood disorders like sickle cell anemia, which is caused by a single gene, could also be treated this way.
"You can envision removing blood cells from a patient, doing the editing and putting those cells back into the patient," Doudna says.