New center provides resources to develop and test new genome editing technologies

Researchers at Baylor College of Medicine and Rice University received a grant for more than $3.9 million over five years from the National Institutes of Health’s Office of Research Infrastructure Programs to establish the Baylor/Rice Genome Editing Testing Center (GETC). The new center will assist investigators from across the country with somatic cell genome editing experiments in mouse models.

Somatic cell genome editing, the ability to edit DNA within the body’s non-reproductive cells, is a promising potential treatment for the most severe human diseases. Over the last decade, significant effort has gone into developing more effective genome editing systems and methods of delivery to specific cells and organs. However, many of these new technologies do not progress to use in humans because there is insufficient evidence from animal models supporting their effectiveness.

“Our center will provide mouse model resources and genome editing testing pipelines to researchers who are developing new genome editing and delivery technologies but need assistance with conducting preclinical animal studies,” said Dr. Jason Heaney, co-principal investigator and associate professor of molecular and human genetics at Baylor. “Our goal is to help generate the animal model data needed to demonstrate the therapeutic potential of these cutting-edge technologies.”    

“We are thrilled to have this opportunity to serve the broader scientific community interested in somatic genome editing. Our goal is to provide a ‘one-stop shop’ for testing in a relevant small animal model. This will allow researchers to answer critical questions about the biodistribution, efficiency, specificity and efficacy of their tools,” said Dr. William Lagor, co-principal investigator and professor of integrative physiology at Baylor. “The aim is to accelerate the clinical translation of new delivery technologies and gene editing systems to treat disease.”

The new center will leverage the multidisciplinary expertise and resources of Baylor’s current NIH-funded projects centered around animal models, such as the Knockout Mouse Phenotyping Project and the Center for Precision Medicine Models. The center will offer paid services, including use of somatic genome editing testing pipelines and mouse models for in vivo testing, to investigators across the country.

“We expect the GETC will make a broad and sustained impact by supporting the development of new genome editing technologies and accelerating their safe and effective deployment to the clinic,” said Dr. Christopher Walkey, co-principal investigator and instructor of integrative physiology at Baylor.

Dr. Denise Lanza, assistant professor of molecular and human genetics at Baylor, will lead mouse resource management. Dr. Cecilia Ljungberg, assistant professor of pediatrics and director of the RNA In Situ Hybridization Core, will supervise and support quantitative tissue imaging.  Dr. Uma Ramamurthy, associate professor of pediatrics and executive director of research information technology in the Office of Research at Baylor, will assist with informatics.

Dr. Gang Bao, department chair and Foyt Family Professor of Bioengineering at Rice, a professor of chemistry, materials science and nanoengineering, and mechanical engineering, and a Cancer Prevention and Research Institute of Texas (CPRIT) Scholar, will lead the efforts in the GETC to determine the efficiency, specificity and safety of genome editing systems in disease models.

“We are very excited to work with Baylor colleagues in the GETC to help researchers test their gene editing approaches,” Bao said. “My lab is well equipped with the tools to analyze gene editing outcomes, including on- and off-target editing in the target tissue, large deletions, vector genome insertions and chromosomal translocations.”