Sickle cell treatment? CRISPR gene editing shows promise

Sickle cell patient. Source: Marcus Yam, Los Angeles Times

A gene editing technology called CRISPR/Cas9 repaired 20 to 40 percent of stem and progenitor cells obtained from sickle cell disease patients, scientists report, suggesting such an approach might offer a way of treating this disease.

Sickle cell anemia is the most severe type of sickle cell disease, which is a group of inherited conditions that affect hemoglobin, the molecule in red blood cells that transports oxygen throughout the body. A single mutation in the gene that helps to make hemoglobin alters red blood cells to a sickle, crescent shape. These cells die prematurely, leading to anemia, and may also get stuck in small blood vessels, blocking blood flow.

Researchers injected these altered cells into the bone marrow of mice with defective immune systems and determined how many retained the edit after 19 weeks. “The rate of repair remained stable, which is great,” [said Gang Bao, PhD, a member of the research team and a bioengineer at Rice.]

Regarding their clinical significance and whether repairing as much as 40 percent of the cells is enough to cure the disease, Bao noted that more work is needed. “We’d like to say, ‘Yes,’” he said, “but we don’t really know yet. That’s something we hope to learn from an eventual clinical trial.”

Read full, original post: CRISPR/Cas9 Gene Editing Shows Potential to Treat Sickle Cell Disease, Study Reports

Outbreak Daily Digest
Biotech Facts & Fallacies
Talking Biotech
Genetics Unzipped
Video: Test everyone – Slovakia goes its own way to control COVID

Video: Test everyone – Slovakia goes its own way to control COVID

As Europe sees record coronavirus cases and deaths, Slovakia is testing its entire adult population. WSJ's Drew Hinshaw explains how ...
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 ...

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