Scientists are developing a genetic tool called CRISPR to repair damaged genes.
Researchers from varying fields of biology are working together to develop a new technique that would allow doctors to go inside cells to make changes to genes. This technique would be faster and inexpensive.
"It's really powerful, it's a really exciting development," said Craig Mello, of the University of Massachusetts Medical School. He was a Nobel Prize winner in 2006 for another technique that can also modify genes. However, Mello says the new tool, called CRISPR, is more powerful "because now you can essentially change a genome at will to almost anything you want. The sky's the limit."
Mello has formed a company called CRISPR Therapeutics to develop innovative therapies for people suffering from genetic blood diseases, like sickle cell and thalassemia. In theory, Mello says it is possible to treat such diseases by removing the bone marrow, repairing the damaged gene, and placing it back in the patient's body. This approach has not yet been possible in practice.
CRISPR's possibilities has many in the scientific world excited about new therapies, but a lot more testing is needed to truly find out if the technique is effective and safe.