New horizons for gene editing

Posted: June 25, 2012 at 10:16 am

Cambridge UK personalised medicines pioneer Horizon Discovery has established three new Centres of Excellence (CoE) for rAAV gene editing.

The Babraham Institute will use Horizons rAAV gene editing technology to understand the different PI3K isoforms and other components of the PI3K signaling networks. PI3K enzymes are critical regulators involved in many cellular responses.

Also through the CoE program, Horizon will support Dana-Farber Cancer Institute and the University of Liverpool in its application of Horizons propriety gene engineering technology, GENESIS, to generate human isogenic cell lines incorporating genes involved in the development of specific diseases, with a focus on cancer.

The Dana-Farber CoE will focus on epigenetic mechanisms involved in the differentiation of normal stem cells and development of normal tissues, and how abnormalities in this process lead to initiation and progression of tumors. They will also develop isogenic cellular models to understand the roles of specific epigenetic genes and their role in breast cancer.

The University of Liverpool is interested the Ras family of proteins (N-, H- and K-Ras) which are involved in many key cellular processes, and how they produce different biological outputs despite their high degree of identity. The university will produce isogenic cellular models that express different forms of these Ras proteins from their endogenous loci.

Dr Rob Howes, principal scientist, Horizon said: We are delighted these three new world class institutes have joined our Centers of Excellence program.

Horizon is hoping to continue expanding the CoE network through 2012, and we look forward to working with an increasing number of excellent scientific groups.

The new human isogenic cell lines generated by the CoEs will be exclusively licensed to Horizon in return for future product royalties. Horizon will also have an exclusive option to license new intellectual property developed.

This forms part of Horizons strategy to generate at least 2500 new X-MANTM (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 rationalise many aspects of drug development, reducing the cost of bringing to market new personalised therapies.

The centers are part of the GENESIS Gene Editing Consortium, which includes rAAV GENESIS pioneers the University of Washington, the University of Torino, Johns Hopkins University, the Translational Genomics Institute (TGen), the University of Minnesota, the University of Maryland, Case Western Reserve University and the University of Pittsburgh.

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New horizons for gene editing

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