Fighting diseases via genome editing

The human body is pretty great, but it could use a few tweaks from time to time.

That's the philosophy of a group of scientists developing a way to edit genomes, a cutting-edge field that could be the answer to many significant diseases and help researchers better understand the human body.

Thanks to these discoveries, scientists can now replace specific parts of the DNA of cells, making a disease weaker or a person less susceptible to the disease, for example. In the case of HIV, which is unable to infect people without a specific protein, genome editing can modify the specific part of the genome so the person no longer produces the protein and the person can no longer be infected with HIV.

"We take out cells, correct it ... and put the cell back into the person," said Feng Zhang, a researcher at the Broad Institute in Cambridge. "It's a biotechnology that allows us to go into the genome, the DNA of a cell, and make very exact changes within the DNA."

It is process that can take weeks, requiring the removal of — in the case of HIV — all of the blood cells so the change can be made to the cells.

Zhang said sickle cell anemia is another disease that could be tackled by genome editing, but any virus — including Ebola — that has a mutation that can be exploited at the genetic level could be addressed by the process.

He is credited with developing CRISPR, a new method of genome editing that his colleagues say made the practice dramatically more widespread.

"Any lab with any biological expertise can do it," said Charles Gersbach of Duke University, who is studying genome editing for genetic diseases.

By breaking the DNA in a specific place, the cell's self-repair mechanism is triggered, but instead of reforming the same DNA sequence, CRISPR provides a new template.

"When you make a cut in the region you want to fix, you can fool the cell into putting in the right information," Zhang said.

Genome editing could also have massive implications for the understanding of the human body. While the human genome has been completely mapped, there are still many aspects that scientists don't understand, Gersbach said.

"We still don't know what most of that sequence means," he said. "You want to treat it like a machine and start disconnecting different parts."

It also could be used to make plants more resistant to diseases or change the immune system in pigs so organs can be used in humans.

"There should be discussion around what can and can't be done," Gersbach said. "That can has been opened, and now we have to figure out what to do with it."

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