4,000 US babies are born with mitochondrial diseases. That could be fixed by a new form of gene editing

michigan charlie gard case x header
5 month old Russell “Bubs” Cruzan III, born with a mitochondrial disease. Credit: Michelle Budnik-Nap

With the first experiments to use CRISPR in people underway, the gene-editing technique is showing promising signs in a few patients. But it turns out not all DNA is amenable to CRISPR.

Some genetic diseases, like those caused by mutations in the genome of the mitochondria — the body’s energy sources — can’t be corrected with CRISPR. [July 8], a team at the Broad Institute of MIT and Harvard and the University of Washington School of Medicine announced that they figured out how to precisely edit mitochondria for the first time.

The discovery could help scientists better understand mitochondrial diseases and test treatments for these disorders, which affect about 1,000 to 4,000 babies born in the United States every year.

Follow the latest news and policy debates on agricultural biotech and biomedicine? Subscribe to our newsletter.
Related article:  Promoting CRISPR crops at the expense of GMOs is short-sighted when we need both

To test the editing system, the researchers used it to make single-letter edits in five different human mitochondrial genes. One of the genes they modified is called MT-ND4, which plays a role in the cell’s energy production processes. When they changed a single C to T, mimicking a mutation, the mitochondria began to break down. Across their experiments, they found that the new method edited about 20% to 40% of the mitochondria that they aimed to change.

“20% to 40% might not sound particularly impressive, but many genetic diseases can be treated by levels of correction that are in that ballpark,” [researcher David] Liu says. “You rarely need to correct 100% of genes to have a benefit to a prospective patient.”

Read the original post

Outbreak
Outbreak Daily Digest
Biotech Facts & Fallacies
Talking Biotech
Genetics Unzipped
can you boost your immune system to prevent coronavirus spread x

Video: How to boost your immune system to guard against COVID and other illnesses

Scientists have recently developed ways to measure your immune age. Fortunately, it turns out your immune age can go down ...
mag insects image superjumbo v

Disaster interrupted: Which farming system better preserves insect populations: Organic or conventional?

A three-year run of fragmentary Armageddon-like studies had primed the journalism pumps and settled the media framing about the future ...
dead bee desolate city

Are we facing an ‘Insect Apocalypse’ caused by ‘intensive, industrial’ farming and agricultural chemicals? The media say yes; Science says ‘no’

The media call it the “Insect Apocalypse”. In the past three years, the phrase has become an accepted truth of ...
globalmethanebudget globalcarbonproject cropped x

Infographic: Cows cause climate change? Agriculture scientist says ‘belching bovines’ get too much blame

A recent interview by Caroline Stocks, a UK journalist who writes about food, agriculture and the environment, of air quality ...
organic hillside sweet corn x

Organic v conventional using GMOs: Which is the more sustainable farming?

Many consumers spend more for organic food to avoid genetically modified products in part because they believe that “industrial agriculture” ...
benjamin franklin x

Are most GMO safety studies funded by industry?

The assertion that biotech companies do the research and the government just signs off on it is false ...
gmo corn field x

Do GMO Bt (insect-resistant) crops pose a threat to human health or the environment?

Bt is a bacterium found organically in the soil. It is extremely effective in repelling or killing target insects but ...
favicon

Environmental Working Group: EWG challenges safety of GMOs, food pesticide residues

Known by some as the "Environmental Worrying Group," EWG lobbies for tighter GMO legislation and famously puts out annual "dirty dozen" list of fruits and ...
m hansen

Michael Hansen: Architect of Consumers Union ongoing anti-GMO campaign

Michael K. Hansen (born 1956) is thought by critics to be the prime mover behind the ongoing campaign against agricultural biotechnology at Consumer Reports. He is an ...
News on human & agricultural genetics and biotechnology delivered to your inbox.
Optional. Mail on special occasions.
Send this to a friend