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 the children’s inner ears called the cochlea, a spiral-shaped chamber that contains hair cells. The first patient in the trial received the gene therapy in … Read more
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 Crispr gene editing. This new therapy was developed by Crispr Therapeutics and Vertex Pharmaceuticals to treat sickle cell disease, an ailment caused by a single-letter … Read more
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 lot of antibodies—thousands of them every second. What if these antibody factories could be harnessed to make other things the body needs? That’s the idea … Read more
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 another cancer has long been considered a theoretical risk. In its notice, the FDA said the use of these viruses may have played a role … Read more
New research seems to show the potential of a CRISPR-based gene therapy meant to neuter certain forms of cardiovascular disease. In the world’s first human trial of the therapy, people given the highest doses experienced significant and long-lasting reductions in their low-density lipoprotein cholesterol (LDL-C). The treatment could be used to help people genetically predisposed … Read more
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 OTOF gene therapy could be used to restore hearing in mice lacking the otoferlin protein. A separate group in Germany published similar findings in 2021, … Read more