Gene, stem and cellular therapies

Theme Lead: Professor Adrian Thrasher

Deputy Theme Lead: Professor Waseem Qasim

This theme aims to develop and refine our state-of-the-art infrastructure to enable us to deliver new gene and cell therapies into the NHS. These therapies use molecular tools to specifically edit faulty cells or genetic material, and replace them with functioning versions. These techniques have previously been used at GOSH to successfully treat infants with childhood cancers such as leukaemia.

During the new funding term we aim to build on these successes and develop similar approaches for a wider range of conditions including areas of unmet need such as skin and muscular diseases.

To achieve this we will be investing in our infrastructure and regulatory processes, as well as working closely with industry partners

Latest research

Learn about the latest research from the Gene, stem and cellular therapies theme

New CAR T-cell therapy for leukaemia associated with less dangerous side effects

A clinical trial to investigate a novel CAR T-cell therapy, designed to target cancer cells more quickly and cause less side effects, has shown very promising results for children with previously incurable acute lymphoblastic leukaemia (ALL).