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AstraZeneca commits to CRISPR gene-editing collaboration

02 February 2015

By Claire Downes

Appeared in BioNews 788

AstraZeneca has joined up with academic and industrial research partners to use CRISPR, a new and much-hyped gene-editing technology, in its quest for new medicines.

The company says it hopes CRISPR will 'accelerate the discovery of novel treatments' for diabetes, cancer and heart disease in particular.

Dr Lorenz Mayr, a vice-president at AstraZeneca, told BBC News that CRISPR represented 'one of the biggest developments in the area of biotechnology in decades'.

CRISPR, which stands for 'clustered regularly interspaced short palindromic repeats', allows scientists to cut out or alter genes of interest in a length of DNA so that they can be investigated in greater detail. The technology relies on two components, an RNA-guide which acts like a homing device to the relevant gene, and an enzyme that cuts the DNA.

CRISPR has become known as a 'genetic scissors' technique but it also allows for greater genetic manipulation including the ability to add nucleotides to the gene sequence.

AstraZeneca's four international partners in the venture all have specific roles to play.

A team at the Wellcome Trust Sanger Institute will focusing on deleting genes relevant to cancer, cardiovascular, respiratory, and autoimmune and inflammatory diseases in order to better understand their role.

The Innovative Genomics Initiative, based at the University of California, USA, will focus on inhibiting and activating genes in order to understand their role in disease pathology and also work on target validation.

Thermo Fisher Scientific, a reagent and instrument company, will provide the RNA-guide libraries that target individual genes and gene families. This will allow AstraZeneca to screen these guides against cell lines to identify new disease targets.

Finally, the Broad Institute and Whitehead Institutes in Cambridge, Massachusetts, USA, will work to evaluate a genome-wide CRISPR library against a panel of cancer cell lines.

Biotechnology industry publication FierceBiotech said the 'ambitious' project represented AstraZeneca's attempt 'to create a new foundation for its once widely ridiculed research and development' operations. The partnership, it said, 'will eventually offer some insight into whether a big corporation can effectively pursue the kind of complex, innovative scientific teamwork needed to actually deliver new programmes for clinical development'.

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