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
US FDA approves Vertex/CRISPR gene therapy for an inherited blood disorder
The headquarters of US biopharmaceutical company Vertex Pharmaceuticals in Boston, Massachusets, on November 4, 2023. Joseph Prezioso | Afp | Getty Images The U.S. health regulator has approved Vertex Pharmaceuticals …
Gene Editing Needs to Be for Everyone
At the end of 2023, we witnessed an important moment in the history of medicine: For the first time, the US Food and Drug Administration approved a therapy that uses …
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 …
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 …
Gene Therapy Targets Early Heart Attack, Stroke Risk – Study
A promising new gene-editing therapy targets heart attack and has shown significant promise in lowering dangerously high cholesterol levels in patients with familial hypercholesterolemia (FH). FH is a genetic disorder …
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
The weight loss was surprising, says Rajagopalan. Ozempic and Wegovy are injected into the fatty tissue of the thighs, waist, or upper arm. From there, it enters the bloodstream, where …