NIH’s Center for Regenerative Medicine (CRM) will engineer stem cells to create isogenic disease models with induced mutations and/or lineage markers using Horizon Discovery’s GENESIS™ genome editing technology, under a technology access agreement by the company.

The 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.

GENESIS uses rAAV vectors to perform gene-editing in human cells through homologous recombination (HR). When harnessed using rAAV gene-editing vectors, HR allows precise alteration of any DNA sequence, allowing more accurate modeling of genetic diseases in human cells, including stem cells, in vitro. While use of the models has been established in oncology, predicting patient responses to targeted therapies both during drug development and in the clinic, Horizon hopes to apply its technology to other scientific areas, enabling development of more personalized medicines.

“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 [Horizon’s Centers of Excellence or] CoE program into stem cell research,” Rob Howes, Ph.D., Horizon Discovery’s CoE program manager, said in a statement.

Under CoE, Horizon commits resources to provide training and open access to GENESIS for academic and not-for-profit research groups or laboratories.

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