A new gene therapy approach aimed at protecting people with type 1 diabetes from developing diabetic kidney disease—a serious ...

A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain ...

Gene therapy is a revolutionary field in modern medicine, offering transformative potential to treat and potentially cure a wide range of genetic and acquired diseases. By addressing the root cause of ...

In May, a historic moment in science and medicine was captured in a single photo that circulated across news outlets ...

Researchers have achieved the first demonstration in mice of using gene therapy to reverse hallmark symptoms of SYNGAP1-related disorder, a devastating condition affecting an estimated 1 million ...

Researchers announce breakthrough results for AMT-130, the first disease-modifying treatment for Huntington's disease, with ...

Genetic deficiency of otoferlin, a protein critical to synaptic transmission by the sensory hair cells of the ear, causes congenital deafness. Medicines to treat the condition are lacking; ...

A new gene therapy can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the amount of blood the heart can pump and dramatically improves ...

UCSF Benioff Children’s Hospital Oakland is enrolling patients in an innovative clinical trial that seeks to cure sickle cell disease. The trial is the first in the U.S. to apply non-viral CRISPR-Cas9 ...

Takeda exits cell and gene therapy development amid cost and safety concerns in Japan Takeda to divest cell and gene assets ...