Vinay Prasad is back at the Food and Drug Administration (FDA), picking up where he left off just two weeks ago. He's in charge of vaccines, gene therapies, and blood products again. The FDA asked him to come back, but it's not clear if he'll keep his other big job as the agency's top medical and science officer. Prasad's sudden exit was a surprise. He left after so...
Mitochondria are like tiny powerhouses inside our cells. They keep us going, but sometimes they go wrong and cause problems. Tiny molecules called miRNAs help control these powerhouses. Scientists have been trying to find ways to see and fix these miRNAs to help treat diseases like cancer. A new idea uses special bubbles, called liposomes, to do this job. These bubb...
In a surprising turn of events, Sarepta Therapeutics has chosen to defy the FDA's request to stop distributing its gene therapy drug, Elevidys. This decision comes after three patient deaths linked to the treatment. The FDA has expressed serious concerns about the safety of the drug and has asked Sarepta to halt all shipments. However, the company has refused, arguin...
In Italy, a big question is being asked about a new treatment for babies born with a rare condition called spinal muscular atrophy (SMA). This condition affects the nerves that control muscles, making it hard for babies to move, breathe, and even swallow. The treatment in question is called onasemnogene abeparvovec (OA), and it's quite different from what doctors hav...
The Duchenne muscular dystrophy community is in turmoil. The death of a second teenager from liver failure has sparked fear and anger. The teenager died after receiving a gene therapy called Elevidys. This therapy is from a company called Sarepta Therapeutics. The teenager's death has left families in a state of uncertainty and desperation. Kameron Lawson re...
A major breakthrough in medicine has occurred. For the first time, doctors have used a custom-made CRISPR-based gene therapy to treat a baby's rare and deadly genetic condition. This isn't just any condition. It's a metabolic disorder that can kill up to half of the children who have it in their infancy. The baby, named KJ, was born last summer with a severe form of ...
Africa faces a major challenge in healthcare. Many people suffer from diseases that could be treated with advanced therapies. These therapies, like cellular and gene treatments, have the power to change lives. They can improve survival rates, enhance quality of life, and reduce side effects. So, why aren't these treatments more widely used in Africa? One big rea...
The world of genetic medicine is advancing rapidly, but not everyone is benefiting equally. People like Emily Kramer-Golinkoff, who has a rare form of cystic fibrosis, are often left behind. This disease, the most common fatal genetic condition in the U. S. , affects around 40, 000 Americans. However, Kramer-Golinkoff's specific genetic mutation means that the medica...
Adeno-associated viruses (AAV) are grabbing attention in the medical world. These tiny viruses are being studied for their potential to treat rare and severe diseases. They are like tiny packages that can deliver genetic material into cells. The outer shell of these viruses, known as the capsid, is made up of three main proteins: VP1, VP2, and VP3. These proteins var...
Lipid nanoparticles, or LNPs, are tiny carriers that can transport genetic material like mRNA and siRNA into cells. They are great for gene therapies because they can cross cell membranes easily, are safe for cells, and can be made in large amounts. However, designing the perfect LNP is tough. It's like trying to solve a puzzle with many pieces that interact in compl...