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NIH to Use Horizon Discovery Gene Editing Tech in Stem Cell Studies

NEW YORK (GenomeWeb News) – Horizon Discovery said today that the National Institutes of Health Center for Regenerative Medicine will use its genome editing technology in disease studies to discover how specific genes affect stem cells.

Under the technology access agreement, NIH CRM will use Horizon's Genesis technology to engineer stem cells to create isogenic disease models with specific induced mutations and lineage markers to understand the effects certain genes have on stem cells as they differentiate.

The Genesis technology uses recombinant AAV (adeno-associated virus) vectors to switch on a natural DNA-repair mechanism called homologous recombination that enables researchers to precisely alter any DNA sequence, which permits accurate modeling of genetic diseases in human cells, including stem cells.

This modelling technology has already been established in oncology to predict patient responses to targeted therapies, Horizon said, adding that the company hopes to expand its use into other areas of personalized medicine research.

“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 [recombinant] AAV to gene target in [embryonic stem] cells, and extends the scope of the [Horizon's Centers of Excellence] program into stem cell research," Rob Howes, Horizon's CoE program manager, said in a statement.

NIH CRM, which was established under the NIH Common Fund to support development of stem cell-based technologies, will use the Genesis technology through Horizon's COE program.

Financial terms of the agreement were not released.

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