Realizing the potential of cell and gene therapies will require organizations to rethink how they finance care and build the ...

Gene therapy is still in development to test its safety and effectiveness, but it may be a promising new treatment for rheumatoid arthritis (RA). Gene therapy alters certain genes to help treat ...

Viral vectors dominate gene therapy, with lentivirus, adenovirus, and AAV being key players, each with unique advantages and limitations. Non-viral vectors, such as lipid nanoparticles and GalNAc, ...

Clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated (Cas) proteins are core components of fast-evolving therapeutic gene editing tools. Scientists have used CRISPR ...

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University’s College of Medicine and the UMass Chan Medical School have achieved ...

An experimental gene therapy was found to slow the progression of Huntington’s disease in a “pivotal” Phase 1/2 study, according to drugmaker uniQure, marking a major step toward a potential first ...

Gene therapy has been effective for young children with genetic hearing loss, but this is the first study to show promising results in older patients. Reading time 3 minutes In recent years, gene ...

Jessica Rendall is a reporter with experience covering a variety of health and wellness topics, including chronic disease, infectious disease, telemedicine, mental health and health technology. Kelly ...

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 ...