A US biotechnology company has tested a gene therapy designed to reverse ageing in humans for the first time. View on ...

A groundbreaking gene-editing therapy developed by Eli Lilly and Verve Therapeutics reduced "bad" LDL cholesterol by up to 62 ...

In the spring of 2024, the US Food and Drug Administration (FDA) approved fidanacogene elaparvovec (Beqvez), only the second gene therapy product for hemophilia B. Just a few months later, the Pfizer ...

In LAMA2-related muscular dystrophy, weakened extracellular matrix (yellow) leads to muscle fiber degeneration and the infiltration of immune cells (red) into the tissue. Researchers at the University ...

LDL Reduction: Studies have found that this therapy successfully reduced LDL cholesterol by up to 62 percent. Let's explain it in detail. Treating bad cholesterol with gene therapy How Gene Therapy ...

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.

Genetic diseases impact almost 70-80 million people worldwide. Oftentimes, there are limited treatments that doctors can provide, leaving patients with few interventions to manage symptoms. Recently, ...

A prenatal gene therapy for hemophilia A showed no detectable maternal exposure to engineered cells or FVIII protein in sheep ...

Four decades have passed since cochlear implants gave infants born deaf the ability to hear. Now, gene therapy promises to restore natural hearing for those born with a rare form of deafness, and the ...

Physician and molecular biologist Katherine High remembers sitting at a staff meeting of the gene therapy company Spark Therapeutics on November 15, 2018, waiting to hear from a guest speaker, when ...