After 30 years of continuing improvements, gene therapy appears to be on the cusp of living up to that promise. Today more than 700 gene therapies including gene-modified cell therapies are being ...

They used the mechanisms zebrafish rely on to repair damage to their hearts combined with viral vectors used for gene therapy in humans. In a new paper appearing online Dec. 13 in Cell Stem Cell, the ...

Zandy Forbes’ biotech company MeiraGTx has developed targeted gene therapy methods to treat more than just genetic disorders. Now it’s on the cusp of its first regulatory approval. Each year ...

Its fall showcases the difficulties that many gene therapy companies are facing in the commercialization of their products. “For more than a decade, Bluebird has been at the forefront of gene ...

Yet on Tuesday, Sarepta shares sank as much as 25% after the company reported the death of a young man from acute liver failure following treatment with its gene therapy. The news was ...

“This is the beginning of a new era of medicine,” said Dr. Kiran Musunuru, a gene therapy researcher at the University of Pennsylvania’s Perelman School of Medicine. He added that the method ...

From reproductive rights to climate change to Big Tech, The Independent is on the ground when the story is developing. Whether it's investigating the financials of Elon Musk's pro-Trump PAC or ...

An experimental trial of gene therapy has helped four toddlers - born with one of the most severe forms of childhood blindness - gain "life-changing improvements" to their sight, according to ...

Tomas has become the first in the world to be treated with a new gene therapy (Handout/PA) A baby boy with a rare illness is making “incredible” progress after he became the first ever patient ...

Scientists have created a new gene therapy for a debilitating genetic disorder called maple syrup urine disease (MSUD). The treatment can prevent recurrence of deadly symptoms in a cow calf born ...

An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment. Colton Belluzzo was diagnosed with a form of muscular dystrophy when ...

Researchers identified a gene that seems to help slow brain aging in women, and studied links between hormone therapy, menopause and Alzheimer’s. By Gina Kolata Women’s brains are superior to ...