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

| | July 23, 2020
michigan charlie gard case x header
5 month old Russell “Bubs” Cruzan III, born with a mitochondrial disease. Credit: Michelle Budnik-Nap
This article or excerpt is included in the GLP’s daily curated selection of ideologically diverse news, opinion and analysis of biotechnology innovation.

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:  Wheat and celiac disease: Modern breeding not to blame for gluten—but gene editing could help

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 Daily Digest

podcasts GLP Podcasts More...
Biotech Facts & Fallacies
Talking Biotech
Genetics Unzipped

video Videos More...
stat hospitalai ink st x mod x

Meet STACI: STAT’s fascinating interactive guide to AI in healthcare

The Covid-19 pandemic underscores the importance of the technology in medicine: In the last few months, hospitals have used AI ...

bees and pollinators Bees & Pollinators More...
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 ...

infographics Infographics More...
breastfeeding bed x facebook x

Infographic: We know breastfeeding helps children. Now we know it helps mothers too

When a woman becomes pregnant, her risk of type 2 diabetes increases for the rest of her life, perhaps because ...

biotechnology worker x

Can GMOs rescue threatened plants and crops?

Some scientists and ecologists argue that humans are in the midst of an "extinction crisis" — the sixth wave of ...
food globe x

Are GMOs necessary to feed the world?

Experts estimate that agricultural production needs to roughly double in the coming decades. How can that be achieved? ...
eating gmo corn on the cob x

Are GMOs safe?

In 2015, 15 scientists and activists issued a statement, "No Scientific consensus on GMO safety," in the journal Environmental Sciences ...
glp profiles GLP Profiles More...
Screen Shot at PM

Charles Benbrook: Agricultural economist and consultant for the organic industry and anti-biotechnology advocacy groups

Independent scientists rip Benbrook's co-authored commentary in New England Journal calling for reassessment of dangers of all GMO crops and herbicides ...
Screen Shot at PM

ETC Group: ‘Extreme’ biotechnology critic campaigns against synthetic biology and other forms of ‘extreme genetic engineering’

The ETC Group is an international environmental non-governmental organization (NGO) based in Canada whose stated purpose is to monitor "the impact of emerging technologies and ...
report this ad report this ad report this ad


News on human & agricultural genetics and biotechnology delivered to your inbox.
Optional. Mail on special occasions.
Send this to a friend