The development of a pioneering gene therapy to treat a devastating, rare immune disorder led by UCL scientists is set to ...
One of the more exciting opportunities in medical technology is using a virus to carry genetic information in the form of DNA and RNA in the cells. One of the most commonly used viruses is ...
Gene therapy holds tremendous promise for treating a wide range of hereditary and acquired diseases by delivering exogenous therapeutic nucleotide sequences into specific cells or tissues. Recent ...
An eight-month-old infant with a severe genetic epilepsy has become the first patient in the world to receive an experimental gene replacement therapy designed to restore the function of the WWOX gene ...
While gene therapy for cystic fibrosis is still in the research phase, researchers aim to correct the defective gene responsible for the disease. Current research is promising. Cystic fibrosis (CF) is ...
Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...
This past spring, a biotech company announced the first use of a new gene-editing technology in people to fix an errant gene that causes a severe immune disorder. In June, a baby born with a ...
Cardiovascular diseases are the leading cause of death globally, with cardiac arrhythmias contributing substantially to this burden. Gene therapy, which directly targets the underlying disease ...
Quality by design is critical for cell and gene therapy manufacturing where process controls directly influence product ...
Morning Overview on MSN
The FDA reversed course and cleared the first gene therapy for Huntington’s disease toward approval
Families affected by Huntington’s disease gained a rare reason for optimism on June 17, 2026, when uniQure N.V. disclosed ...
Diseases that affect the retina, the light-sensitive layer at the back of the eye, are a significant cause of visual impairment and blindness. Gene therapy holds promise for treating some of these ...
Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.
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