More quality-adjusted life-years with gene therapy, but at a much higher cost ...

A cutting-edge procedure for treating sickle cell disease is changing lives in the St. Louis area, where an estimated 1,200 ...

An eight-month-old infant with a severe genetic epilepsy has become the first patient in the world to receive an experimental gene replacement therapy designed to restore the function of the WWOX gene ...

Muscle spasticity is a common and often debilitating consequence of spinal cord injury that can significantly affect mobility ...

An 8-month-old infant with severe genetic epilepsy has become the first patient in the world to receive an experimental gene ...

UCSD study says the therapy increased expression of a neuroprotective protein to help preserve brain functions, despite the ...

Emerging gene editing platforms are revolutionizing precision medicine, enabling safe and effective genetic modifications for ...

Otarmeni, a surgically-delivered drug developed by Regeneron, recently received accelerated FDA approval to treat a rare, ...

Revolutions in gene therapy are rapidly changing the landscape of modern medicine. Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike ...

Gene editing is now reaching the mainstream, ushering in a new era of genetic manipulation. Traditionally, inserting or deleting entire genes, regulating their expression, and altering specific ...

Genetic manipulation of malaria parasites has transformed our understanding of Plasmodium biology and accelerated the search for novel interventions. Early approaches relied on homologous ...