When CRISPR gene editing falls short: ‘Base editing’ might treat disorders on the maternal line in mitochondrial DNA

download
Credit: Angela Gao

[A] technique — which builds on a super-precise version of gene editing called base editing — could allow researchers to develop new ways to study, and perhaps even treat, diseases caused by mutations in the mitochondrial genome. Such disorders are most often passed down maternally, and impair the cell’s ability to generate energy. Although there are only a small number of genes in the mitochondrial genome compared with the nuclear genome, these mutations can particularly harm the nervous system and muscles, including the heart, and can be fatal to people who inherit them.

But it has been difficult to study such disorders, because scientists lacked a way to make animal models with the same changes to the mitochondrial genome. The latest technique marks the first time that researchers have made such targeted changes, and could allow researchers to do this.

Related article:  Video: Inside the quest to improve farm animal welfare by tweaking their genes
Follow the latest news and policy debates on agricultural biotech and biomedicine? Subscribe to our newsletter.

The work is a long way from being used in the clinic, [chemical biologist David] Liu cautions. Although his team’s initial studies found few off-target DNA changes — a common problem in CRISPR–Cas9 gene-editing — more studies in different cell types are needed, he says.

The technique could ultimately complement existing methods used to prevent or treat mitochondrial disorders. Some countries already allow a procedure called mitochondrial replacement, in which the nucleus of an egg or embryo is transplanted into a donor egg or embryo that contains healthy mitochondria.

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