A new gene therapy has been used to successfully treat a deadly childhood liver disease using mice that model this disease by researchers at UCL and Great Ormond Street Hospital.

For some people, cancer immunotherapies are life-changing. These treatments can turn the body’s own immune system against a tumor, either eliminating it or shrinking it enough to make surgery possible. But these therapies don’t work for everyone, can gradually stop working or can come with a host of side effects, including severe inflammation. A new experimental therapy aims to change that. Scripps Research scientists are testing SAR’877, a bispecific molecule designed to restore immune cells’ ability to fight cancer. 

Oral combination therapy demonstrates high response rates for patients with hard-to-treat acute myeloid leukemia

Research brings new biological understanding of cancer cell origin and evolution, age-related inflammation

Studies provide insights into the tumor immune microenvironments of brain metastases treated with radiation therapy and of muscle-invasive bladder cancer

New gene therapy successfully delivers large proteins and restores muscle function in models of muscular dystrophy