Treatment introduces an expansion of the scope of gene therapies that might lead to similar treatments for neurodegenerative diseases in other parts of the body ...

In a preclinical study, UC Davis Comprehensive Cancer Center scientists have developed a highly targeted gene therapy that could revolutionize treatment for cancers linked to a common herpesvirus, ...

After years of heartbreak, researchers have found an experimental treatment that can slow the progression of Huntington’s disease, according to early results from a small clinical trial ...

A gene-editing therapy led to durable changes in serum transthyretin levels among people with hereditary transthyretin amyloidosis with polyneuropathy. Polyneuropathy stages and disability scores were ...

Groundbreaking study demonstrates that targeted oligodendrocyte gene therapy leads to decreased N-acetylaspartate (NAA) levels and increased ...

Share on Pinterest The new gene therapy showed promise by significantly slowing down Huntington’s disease. Santi Nuñez/Stocksy Huntington’s disease is a genetic brain disorder that causes symptoms ...

The first participant in an investigational gene therapy trial of STXBP1 encephalopathy died, Capsida Biotherapeutics said. The death occurred days after dosing, according to information posted on the ...

A team led by scientists at Harvard Medical School, Massachusetts General Hospital, and the Broad Institute of MIT and Harvard used CRISPR technology to pinpoint genes that could be edited to boost ...

New study data showed treatment slowed signs of disease progression by 75% after three years, a finding that could have “massive effects on patients’ lives,” an investigator said.

A child enrolled in Capsida Biotherapeutics’ gene therapy trial for STXBP1 developmental and epileptic encephalopathy has died, prompting the company to pause the study while investigating the death.

The first study to evaluate both eyes treated with a gene therapy targeting neovascular, or wet, age-related macular degeneration (AMD) has found the treatment as safe and effective in the second eye ...