Research on genome editing poses dilemma

Washington - The hottest tool in biology has scientists using words like revolutionary as they describe the long-term potential: wiping out certain mosquitoes that carry malaria; treating genetic diseases like sickle-cell or preventing babies from inheriting a life-threatening disorder.

It may sound sci-fi, but research into genome editing is booming. So is a debate about its boundaries, what's safe and what's ethical to try in the quest to fight disease.

Desperate families

Does the promise warrant experimenting with human embryos? Researchers in China already have and they're poised to in Britain.

Should we change people's genes in a way that passes traits to future generations? Beyond medicine, what about the environmental effects if, say, altered mosquitoes escape before we know how to use them?

"We need to try to get the balance right," said University of California, Berkeley, biochemist Jennifer Doudna. She helped develop new gene-editing technology and hears from desperate families, but urges caution in how it's eventually used in people.

The US National Academies of Science, Engineering and Medicine will bring international scientists and regulators together in December to start determining that balance. The biggest debate is whether it ever will be appropriate to alter human heredity by editing an embryo's genes.

Stronger immune cells

"This isn't a conversation on a cloud," but something that families battling devastating rare diseases may want, Dr George Daley of Boston Children's Hospital told specialists meeting to plan the ethics summit.

"There will be a drive to move this forward."

Laboratories worldwide are embracing a technology to precisely edit genes inside living cells - turning them off or on, repairing or modifying them - like a biological version of cut-and-paste software. Researchers are building stronger immune cells, fighting muscular dystrophy in mice and growing human-like organs in pigs for possible transplant.

Biotech companies have raised millions to develop therapies for sickle cell disease and other disorders.