Skip to main content
Premium Trial:

Request an Annual Quote

In Person Editing

Scientists earlier this week used gene editing within a patient to try to treat his metabolic disease, the Associated Press reports.

The patient, 44-year-old Brian Madeux has Hunter syndrome, an X-linked recessive condition marked by the absence of an enzyme that breaks down certain carbohydrates. Currently, Madeux has been receiving weekly doses of the missing enzyme, which the AP notes can run between $100,000 and $400,000 a year and only eases some symptoms.

Doctors at the University of California, San Francisco's Benioff Children's Hospital infused Madeux Monday with viruses containing corrected copies of the affected gene and the molecular tools to replace the diseased copy, the AP adds. But rather than rely on the CRISPR/Cas9 gene-editing tool, the AP says that scientists here have used zinc finger proteins as the editing machinery. It also notes that gene therapy raises a number of safety concerns, though the researchers say they are using a safer viral vector and that the effects should be limited to the liver.

To successfully treat the disease, study leader Paul Harmatz tells the AP that only about 1 percent of liver cells would have to be altered.

"It's kind of humbling," says Madeux of being the first to undergo this treatment, according to the AP. "I'm willing to take that risk. Hopefully it will help me and other people."

The Scan

ChatGPT Does As Well As Humans Answering Genetics Questions, Study Finds

Researchers in the European Journal of Human Genetics had ChatGPT answer genetics-related questions, finding it was about 68 percent accurate, but sometimes gave different answers to the same question.

Sequencing Analysis Examines Gene Regulatory Networks of Honeybee Soldier, Forager Brains

Researchers in Nature Ecology & Evolution find gene regulatory network differences between soldiers and foragers, suggesting bees can take on either role.

Analysis of Ashkenazi Jewish Cohort Uncovers New Genetic Loci Linked to Alzheimer's Disease

The study in Alzheimer's & Dementia highlighted known genes, but also novel ones with biological ties to Alzheimer's disease.

Tara Pacific Expedition Project Team Finds High Diversity Within Coral Reef Microbiome

In papers appearing in Nature Communications and elsewhere, the team reports on findings from the two-year excursion examining coral reefs.