A new study describes a gene therapy strategy that uses the brain's own glymphatic transport system to distribute engineered ...

Gene therapy represents one of medicine’s most ambitious attempts to treat disease at its root cause by altering the genetic code itself. The approach works by recovering the functions of critical ...

An innovative method that uses modified versions of a bacterial virus effective at delivering treatments to human cells shows promise as a more inexpensive and efficient way to treat some deadly ...

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, ...

Children and young adults born completely deaf because of mutations in the OTOF gene have gained measurable hearing after ...

A new study describes a gene therapy strategy that uses the brain's own glymphatic transport system to distribute engineered viral vectors throughout the brain.

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

Children born profoundly deaf because of mutations in the OTOF gene are hearing speech, music, and everyday sounds after a single gene therapy infusion, and the gains are holding steady for up to 2.5 ...

Despite the best efforts of modern medicine, Huntington’s disease is a condition that still comes with a tragic prognosis. Primarily an inherited disease, its main symptoms concern degeneration of the ...

Gene therapy achieved its first major breakthroughs in the early 1990s with the treatment of a rare disease called severe combined immunodeficiency. However, the field was dealt a serious setback in ...