Scientists Successfully Edit Human DNA to Eliminate Genetic Defect

In a groundbreaking project carried out by American scientists, human genes have been successfully mutated for the first time. This editing of a disease-causing gene to produce a healthyn embryo is a remarkable milestone. It will provide hope for parents who want to protect their babies from hereditary and other diseases.

This experiment helped to eliminate a dangerous and common gene defect known as hypertrophic cardiomyopathy which causes otherwise healthy people to die all of a sudden due to a heart attack.

In about two third of the embryos – 42 out of 58 – the heart disease was gotten rid off by editing the DNA of the sperm used to create them, a team reported in the Nature journal.

Newly fertilized eggs before gene editing (L) and embryos after gene editing & few rounds of cell division (R)

“We’ve always said in the past gene editing shouldn’t be done, mostly because it couldn’t be done safely. That’s still true, but now it looks like it’s going to be done safely soon,” said Richard Hynes, a cancer researcher at MIT.

This mutation will not only keep the human resulting from the embryo safe from the disease but will also be hereditary and could be passed on to future generations resulting in more  disease-free, healthy babies.

A lot of further research still needs to be done before testing this clinically which is not permissible under Federal Law. The experiment can be applied on any of more than 10,000 conditions caused by specific inherited mutations including breast and ovarian cancer linked to BRCA mutations, as well as diseases like Huntington’s, Tay-Sachs, beta thalassemia, and even sickle cell anemia, cystic fibrosis or some cases of early-onset Alzheimer’s.

“You could certainly help families who have been blighted by a horrible genetic disease,” said Robin Lovell-Badge, a professor at Francis Crick Institute in London.

Regulatory issues

This will also face a lot of regulatory hurdles before clinical experimenting. There are a lot of questions and a lot of controversy regarding whether this is ethical or not and how it will be regulated.

Critics from around the globe

“If irresponsible scientists are not stopped, the world may soon be presented with a fait accompli of the first [genetically modified] baby. We call on governments and international organizations to wake up and pass an immediate global ban on creating cloned or GM babies, before it is too late,” says David King, the head of Human Genetics Alert, UK.

However, there are many scientists who take it as a great step ahead and would like to take the research forward.

“It’s a pretty exciting piece of science. It’s a technical tour de force. It’s really remarkable,” says George Daley, dean of the Harvard Medical School.

“I think this needs to be tightly regulated. This is very exciting. But it also could be a double-edged sword. So I think we really have to be extra cautious with this technology,” says Fredrik Lanner, a geneticist at the Karolinska Institute in Stockholm.

Although human embryo related experiments are not permitted in the US, the genetic experiments are permitted in the UK.

“If other countries would be interested, we would be happy to work with their regulatory bodies,” says Shoukhrat Mitalipov, director of the Oregon Health & Science University’s Center for Embryonic Cell and Gene Therapy.

via NYTimes