First gene-editing experiments on adults suffering from rare metabolic disorders offer promising early results

genetherapy x
Brian Madeux, who has Hunter syndrome, received a treatment aimed at editing the genome of his liver cells. Image credit: AP Photo/Eric Risberg

In late 2017, scientists first began attempting to edit the genes of adults to treat rare genetic disorders. Preliminary results from a clinical trial, shared by the researchers at the WORLDSymposium conference in Orlando, Florida [on February 7] suggest success in actually altering the DNA, although it’s still unclear whether the intervention will help the patients.

The men enrolled in the studies by the company Sangamo include eight patients with Hunter syndrome and three with Hurler syndrome, according to the AP. These are rare metabolic disorders caused by mutations that leave the men without an enzyme needed to break down certain polysaccharides. In turn, sugars accumulate in the body, leading to organ damage and often early death.

Related article:  130 researchers urge Europe not to banish crop gene-editing with 'strict' regulations

Scientists are using zinc finger nucleases delivered by a virus to cut the defective gene at specific locations and replace it with a corrected code.

A few of the Hunter patients experienced a boost in the level of the missing enzyme, but none reached the typical level of unaffected people. Preliminary results from those treated for Hurler syndrome showed an uptick to normal levels.

“It looks like it’s safe . . . that’s a very positive sign,” [said] Kiran Musunuru.

Read full, original post: Preliminary Results Point to Success of In Vivo Gene Editing

Outbreak
Outbreak Daily Digest
Biotech Facts & Fallacies
Talking Biotech
Genetics Unzipped
Infographic: The evolutionary history of the COVID-19 coronavirus

Infographic: The evolutionary history of the COVID-19 coronavirus

Reuters analysed over 185,000 genome samples from the Global Initiative on Sharing All influenza Data (GISAID), the largest database of ...
favicon

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

Known by some as the "Environmental Worrying Group," EWG lobbies ...
m hansen

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

Michael K. Hansen (born 1956) is thought by critics to be ...
News on human & agricultural genetics and biotechnology delivered to your inbox.
glp menu logo outlined

Newsletter Subscription

Optional. Mail on special occasions.
Send this to a friend