Two teams of researchers from Harvard University have come up with a new gene editor that makes use of the revolutionary CRISPR-Cas9 technology to edit human genes. The breakthrough studies offer new hope for treating, as well as curing, numerous types of genetic or inherited diseases like inherited blindness and cystic fibrosis among others.
One research team changed DNA’s order of atoms to rewrite the human genetic code, thereby being able to alter the instructions for life. While, the other team of scientists edited DNA’s chemical cousin — called RNA, which unlocks the genetic code’s information. Incidentally, all the experiments were conducted on human cells growing in the lab.
The first team of researchers created tools called base editors that can change the molecular structure of one base to change it into another. Consequently, scientists can now control the four bases that make DNA — namely, thymine, guanine, cytosine and adenine.
While conducting the research, the team took the help of base editing to rectify a genetic disease that causes potent levels of iron in the blood. The base editing tool has the potential to rectify 14 percent of human diseases related to a single-letter mutation. Therefore, the tool will help scientists address 48 percent more of these kinds of diseases.
The DNA has to create an RNA copy for a cell to utilize genetic instructions, though the DNA is the genetic code’s master copy. The scientists used their RNA approach to work on an inherited type of anemia in human cells.
The researchers hope to turn certain cells’ protein-production on and off without creating permanent changes to the genetic code of a person. A technique like this has the potential to treat diseases of the kidney, liver, muscle, brain, as well as autoimmune disorders and cancer.
"The ability to correct disease-causing mutations is one of the primary goals of genome editing,” scientist Feng Zhang said. "This new ability to edit RNA opens up more potential opportunities to... treat many diseases, in almost any kind of cell."
However, though the studies and their results are promising, Liu has said that base editors will not be used to treat genetic diseases in living humans in the near future. Scientists still have to figure out the best way to deliver the base editor machinery to the correct tissues in the body and subsequently into the correct cells.
Moreover, the researchers have to also understand what time is the best in a patient’s life to deliver a certain gene therapy. At present, Liu’s lab is working with other labs that have expertise in genetic diseases.