US stem cell coup for Horizon

Technology from Cambridge UK personalised medicines pioneer, Horizon Discovery, is being used for a major stem cell project in the US.

The agreement will enable the NIH CRM to apply Horizons GENESIS genome editing technology to engineer stem cells in order to create isogenic disease models with induced mutations and/or lineage markers.

These disease models will be used to help researchers understand the effects of specific genes and mutations as stem cells differentiate, as well as create lineage reporters for stem cell differentiation.

The NIH CRM was recently established through support from the NIH Common Fund to provide the infrastructure to support and accelerate the clinical translation of stem cell-based technologies, and to develop widely available resources to be used as standards in stem cell research.

The GENESIS technology harnesses rAAV vectors to perform accurate and efficient gene-editing functions in human cells, by switching on a natural high-fidelity DNA-repair mechanism called homologous recombination (HR).

When harnessed using rAAV gene-editing vectors, HR allows the precise alteration of any DNA sequence, permitting the accurate modelling of genetic diseases in human cells, including stem cells, in vitro.

The use of these models in oncology is well established, and has predicted patient responses to targeted therapies both during drug development and in the clinic. By applying this technology to other scientific areas Horizon hopes to lead to the development of personalised medicines by advancing scientific research.

Through our CoE program, we aim to work with world leading experts in their fields, so we are delighted to be working with the NIH CRM, a key centre for stem cell research, said Dr Rob Howes, Horizons CoE Program Manager.

This agreement builds on proof of concept work carried out by Horizon Scientific Advisory Board member and rAAV-mediated gene editing inventor, David Russell, demonstrating the ability of rAAV to gene target in ES cells, and extends the scope of the CoE program into stem cell research.

Horizons CoE program encompasses academic and not-for-profit research groups or laboratories to which Horizon commits resources to provide training and open access to its proprietary rAAV-mediated human gene-editing platform, GENESIS.

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US stem cell coup for Horizon