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Read all about gene therapy including current medical research on switching therapeutic genes on and off, light-activated gene therapy and gene silencing.
In a landmark decision, the Food and Drug Administration Friday approved the first gene-editing treatment to alleviate human illness. The FDA approved two gene therapies for anyone 12 and older...
Today, the U.S. Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease...
Gene therapy involves the introduction of new genes into cells, to restore or add gene expression, for the purpose of treating disease. Most commonly a mutated gene is replaced with DNA...
The FDA on Friday also approved a second treatment for sickle cell disease, called Lyfgenia, a gene therapy from drugmaker Bluebird Bio. Both treatments work by genetically modifying a...
On Wednesday, Kendric Cromer, a 12-year-old boy from a suburb of Washington, became the first person in the world with sickle cell disease to begin a commercially approved gene therapy that...
The Food and Drug Administration approved the first gene therapy for Duchenne muscular dystrophy, but limited access to those ages four and five.
The US Food and Drug Administration approved two gene-based treatments for sickle cell disease Friday, including the first therapy that uses the gene-editing technique CRISPR, opening a new era...
Nature Biotechnology - New drug approvals reached an all-time high in 2023, with five gene therapies, the first CRISPR–Cas9-edited therapy and a disease-modifying Alzheimer’s drug.
The UK’s regulator has approved the world’s first CRISPR–Cas9 gene editing therapy, which aims to cure sickle cell disease and transfusion-dependent β-thalassemia.