A research team has successfully demonstrated the world's first gene-editing treatment for Leber's hereditary optic ...

Gene therapy holds the promise of preventing and curing disease by manipulating gene expression within a patient's cells. However, to be effective, the new gene must make it into a cell's nucleus. The ...

Groundbreaking advancements in gene therapy at UTMB represent new possibilities for patients living with rare genetic ...

Gene therapy holds the promise of preventing and curing disease by manipulating gene expression within a patient's cells. However, to be effective, the new gene must make it into a cell's nucleus. The ...

One of the assets shelved by Eli Lilly is a gene therapy for dementia, which it obtained in its $1.04-billion acquisition of Prevail Therapeutics in late 2020.

The FDA has cleared the first human trial of an age-rejuvenating gene therapy, ER-100, to test safety and vision outcomes ...

When it comes to fighting COVID-19, scientists are throwing every bit of science we’ve got at it. A creative technique some ...

Vertex executives warned that Casgevy, its curative treatment for sickle cell disease, would be slow to reach patients. But ...

NTLA stock faces binary risk: success could unlock multi-billion dollar markets, while setbacks may erase hundreds of ...

A research team led by Professor Hyunji Lee of the Department of Convergence Medicine at Korea University College of Medicine (co-first authors: PhD candidates Sanghun Kim, Ji Eun Kim, and Sungjin Ju; ...

A TOP NHS doctor blasted health advisers for refusing to sanction a 17p test that screens newborns for a horrific disease ...

The FDA has placed clinical holds on a pair of investigational gene therapies developed to address two rare neurodevelopmental disorders in children, according to the manufacturer. In a press release, ...