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Horizon and NIH CRM Collaborate to Apply rAAV Genome Editing to Stem Cells

Published: Friday, October 05, 2012
Last Updated: Friday, October 05, 2012
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Collaboration aims to understand the effects of specific gene sequences on stem cell differentiation.

Horizon Discovery (Horizon) has announced that it has signed a technology access agreement with the U.S. National Institutes of Health Center for Regenerative Medicine (NIH CRM), through Horizon’s Centers of Excellence (CoE) program.

The agreement will enable the NIH CRM to apply Horizon’s 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 modeling 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 personalized 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, CoE Program Manager, Horizon Discovery.

Dr Howes continued, “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.”

Horizon’s 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™.

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

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