6 Deaf Children Can Now Hear After a Single Injection
To get the new genetic material into cells, they engineered harmless viruses to carry it. Doctors carefully injected a tiny amount of liquid containing the viruses into a part of …
gene therapy
In a World First, a Patient’s Antibody Cells Were Just Genetically Engineered
Our B cells help prevent us from getting sick. Their job is to make antibodies, immune system proteins that fight off viruses and other foreign invaders. And they make a …
FDA approves 2 gene therapies for sickle cell disease – National
The U.S. Food and Drug Administration (FDA) on Friday approved a pair of gene therapies for sickle cell disease, including the first treatment based on the breakthrough CRISPR gene editing …
A Cutting-Edge Cancer Treatment May Cause Cancer. The FDA Is Investigating
Scientists use harmless viruses to ferry and insert the new genetic material because of their natural ability to get inside cells. But the potential for these viruses to accidentally trigger …
New Trials Aim to Restore Hearing in Deaf Children—With Gene Therapy
While the Novartis trial was happening, Lustig and others were working on ways to restore hearing in genetic forms of hearing loss. In 2019, Lustig and his collaborators showed that …
Injecting a Gene Into Monkeys’ Brains Curbed Their Alcohol Use
When they conducted postmortem examinations of the monkeys’ brains, the team also confirmed that the treated animals had replenished levels of dopamine. In the untreated animals, dopamine levels remained low. …
A One-Time Shot for Type 2 Diabetes? A Biotech Company Is On It
So far, Fractyl has only tested this approach in animals. Scientists at the company wanted to see how well the experimental gene therapy could reduce fasting blood sugar—an indicator used …