Precision Genome Editing Enters the Modern Era: Can We Cure Genetic Diseases by Rewriting DNA?

CRISPR has sparked a renaissance in genome editing. Now, next-generation CRISPR technologies let scientists modify the genome more efficiently and precisely than before. Such tools could one day serve as therapeutics, but many challenges remain.

 

Most drugs are small molecules that can be packaged into a pill. Genome editors are large, complicated molecules – so scientists can’t just stuff them into a pill for people to swallow, or inject them into people’s bodies. They have to find other ways to get the molecules into patients’ cells. One method relies on viruses, says Guangping Gao, a gene therapy researcher at the University of Massachusetts Medical School and president of the American Society of Gene and Cell Therapy. Scientists could potentially package genome editors into small viruses like adeno-associated viruses, for example. These viruses, which have already seen clinical use in several FDA-approved drugs, could then infect patients’ cells and dump their payloads.

 

It could be that scientists will need to develop entirely different delivery systems. Researchers are currently experimenting with lipid nanoparticles and using electric fields to coax genome editors into cells that can then be transplanted into patients. Delivery remains a major hurdle, Gao says, but he’s still excited about genome editors’ potential. “Gene therapy is now in its golden age,” he says. And genome editors “open even more avenues for treating disease.”

Read the full article at: www.hhmi.org