WASHINGTON DC: A new study in mice suggests that replacing a dysfunctional gene may improve survival in some people with arrhythmogenic right ventricular cardiomyopathy (ARVC), a rare inherited disorder in which the heart’s muscular walls gradually weaken, putting patients at risk of dangerous irregular heartbeats.The investigational treatment targets the loss of function of a gene … Read more
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
Gene therapy has allowed several children born with inherited deafness to hear. A small study published Wednesday documents significantly restored hearing in five of six kids treated in China. On Tuesday, the Children’s Hospital of Philadelphia announced similar improvements in an 11-year-old boy treated there. And earlier this month, Chinese researchers published a study showing … 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
Gautam Dongre’s two children in India and Pascazia Mazeze’s son in Tanzania live with an inherited blood disorder that turns blood cells into instruments of pain. New gene therapies promise a cure for sickle cell disease, and Dongre says he’s “praying the treatment should come to us.” But experts say the one-time treatment is out … Read more
Gautam Dongre’s two children in India and Pascazia Mazeze’s son in Tanzania live with an inherited blood disorder that turns blood cells into instruments of pain. Now that new gene therapies promise a cure for their sickle cell disease, Dongre says he’s “praying the treatment should come to us.” But experts say the one-time treatment … Read more
New Delhi: The US Food and Drug Administration (USFDA) last week approved two breakthrough gene therapies — Casgevy by Vertex Pharmaceuticals and CRISPR Therapeutics, and Lyfgenia by Bluebird Bio — for sickle cell disease (SCD) in patients 12 years and older. The development marks a milestone medical advancement in treating a debilitating disease that primarily affects red … Read more
Regulators on Friday approved two gene therapies for sickle cell disease that doctors hope can cure the painful, inherited blood disorder that afflicts mostly Black people in the U.S. The Food and Drug Administration said the one-time treatments can be used for patients 12 and older with severe forms of the disease. One, made by … Read more
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 technology. The agency approved Lyfgenia from bluebird bio BLUE.O, and a separate treatment called Casgevy by partners Vertex Pharmaceuticals VRTX.O and CRISPR Therapeutics CRSP.BN. Both the therapies were approved for people … 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