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...
Nanopore sensors have emerged as highly sensitive tools, capable of detecting individual particles by measuring changes in ionic current. This method is both straightforward and effective, allowing scientists to gather detailed information about particles without needing any labels. It is a big deal in the world of science. The architecture of nanopores has seen sig...
Retinitis pigmentosa (RP) is a leading cause of inherited blindness globally. It causes a slow but steady loss of both rod and cone photoreceptor cells. These cells are crucial for seeing in the dark and during the day, respectively. Most RP-causing genes only affect rods, but cones also die off later. This secondary cone death might be due to metabolic issues. Phot...
A recent event has brought attention to the risks of gene therapy for Duchenne muscular dystrophy. A 16-year-old boy lost his life after receiving Elevidys, a treatment designed to combat this severe condition. This unfortunate incident marks the first known death associated with this particular therapy. The teenager's passing was due to liver failure, a complication...