Dravet syndrome and other developmental epileptic encephalopathies are rare but devastating conditions that cause a host of ...

Gene editing is already being used to treat a handful of rare genetic diseases; however, most therapies involve breaking or ...

A few examples of immune checkpoint inhibitors include: Gene therapy is a way of treating diseases by editing or altering the genes within the cells of your body. In the case of cancer ...

The majority are in the very early preclinical stage of development, but applications for regulatory approval have been ...

Cell and gene therapies can be life-changing for patients with certain conditions, but the process of receiving them poses barriers for patients and caregivers that require multistakeholder solutions, ...

“You are one of the few in the country and even in the world, you know, taking this big step — gene therapy,” said Campbell, director of the hospital’s Comprehensive Sickle Cell Disease ...

The California Institute for Regenerative Medicine has awarded a $6 million grant to USC investigators pioneering a new first-of-its-kind genetic therapy for glioblastoma, a severe form of brain ...

Gene therapy has been available since 2022 for hemophilia B and since 2023 for hemophilia A, yet some major medical institutions have barely treated any patients so far. What’s the holdup?

Similar targeted genetic therapy approaches have been evaluated ... with predicted biological effect within tumor cells, for example cytotoxic-induced tumor cell death due to the activated ...