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Universal genome editing strategies for ataxia telangiectasia

   School of Biological Sciences

About the Project

Ataxia telangiectasia (A-T) is a severe autosomal recessive genetic disease caused by mutations in the ATM gene, which lead to cerebellar degeneration, cancer susceptibility and immunodeficiency. ATM is a large gene spanning 66 exons, constitutively expressed in all cells. ATM encodes the apical ATM kinase, which plays a central role in the cellular response to DNA double strand breaks (DSBs), apoptosis and cell-cycle checkpoint control. No effective treatment is currently available for A-T, but correction of the mutated gene through CRISPR/Cas genome editing is feasible. However, mutations in ATM are spread across the whole gene, with little evidence of hot-spots, complicating the development of gene correction approaches of wide applicability. We are interested in the design of such universal strategies to correct ATM, based on the replacement of large segments of the gene through genome editing. This project will design, test and optimise CRISPR/Cas genome editing approaches able to correct multiple mutations in ATM. The project will involve the use of advanced genetic engineering, viral vectors (integration-deficient lentiviral vectors and adeno-associated viral vectors), cell lines and haematopoietic stem cells. The work will be done in our Royal Holloway University of London laboratory (http://AGCTlab.org), which is focused on the development of new gene and stem cell therapies for rare and common diseases, and may also involve colleagues in other institutions.

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