Gene editing to model Huntington's disease

The UCL Institute of Neurology has entered into a research collaboration for gene editing with Horizon Discovery (Horizon), a leading provider of research tools to support the development of personalized medicines.

The program of research will focus on the creation of human isogenic disease model cell lines with insertion of Huntington's disease-causing triplet repeats.

The UCL collaboration contributes to an academic not-for-profit research group to which Horizon commits resources to provide training and access to its proprietary rAAV-mediated human gene-editing platform, GENESIS™. The program at UCL represents a new disease area for Horizon, and the insertion of triplet repeats into a wild type genome is a novel genetic alteration application for the GENESIS technology.

Sarah Tabrizi, Professor of Clinical Neurology at the UCL Institute of Neurology, global Principal Investigator of the TRACK-HD study and leader of all UCL Huntington's disease projects, said: "Although mouse models have given important insights, it is important when studying human disease to use a broad approach, including cultured cells. The ability to accurately introduce the triplet repeat mutation into human somatic cell lines offers exciting possibilities in the study of Huntington's disease and potential therapies.

"The use of patient-relevant disease models created by Horizon's rAAV-mediated genome editing technology is well established in oncology," commented Dr Rob Howes, Principal Scientist, Horizon Discovery. "We believe that by developing the application of this technology to other disease areas such as Huntington's disease, we can provide a vital tool for understanding, preventing and treating those diseases."

The new human isogenic cell lines generated by UCL will be exclusively licensed to Horizon. Horizon will also have an exclusive option to license new intellectual property developed. This forms part of Horizon's strategy to generate at least 2500 new X-MAN™ (gene X- Mutant And Normal) models of cancer, neurodegenerative, and cardiovascular disease. These models support drug discovery researchers in their efforts to understand how complex genetic diseases manifest themselves in real patients, and help rationalize many aspects of drug development, reducing the cost of bringing to market new personalized therapies.

The UCL collaboration will complement the GENESIS Gene Editing Consortium, which includes rAAV GENESIS pioneers the National Cancer Institute, Cambridge University, Yale University, and Dana-Farber Cancer Institute.

Horizon recently launched an online support site for scientists working with rAAV-mediated genome editing.

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Links:

• Horizon Discovery