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New Gene and Cell Therapy program launches in pediatric research


A new research program in gene and cell therapy has launched within the Herman B Wells Center for Pediatric Research<~ ink.aspx?_id="92302C6E9AFF496F9C96F9952B50ABB7&_z=za"> at Indiana University School of Medicine<~ ink.aspx?_id="92302C6E9AFF496F9C96F9952B50ABB7&_z=za">. Directed by Roland Herzog, PhD<~ ink.aspx?_id="92302C6E9AFF496F9C96F9952B50ABB7&_z=za">, the Gene and Cell Therapy<~ ink.aspx?_id="92302C6E9AFF496F9C96F9952B50ABB7&_z=za"> program seeks to improve current treatments, develop corrective therapies and explore the use of new technologies to address genetic diseases.<~ ink.aspx?_id="92302C6E9AFF496F9C96F9952B50ABB7&_z=zp">

The primary focus of the research group is to examine the interaction between gene therapies and the immune system. By examining these therapies in hemophilic models, Herzog and his team have already been successful in reversing the disease and hope to address the issue of immune rejection. While the group uses hemophilia as a model, progress in their discovery has major implications for other diseases such as diabetes, asthma and cancer.<~ ink.aspx?_id="92302C6E9AFF496F9C96F9952B50ABB7&_z=zp">

“I am especially excited that Dr. Herzog’s group has the potential to provide technologies that augment the work of each program within the Wells Center,” says Wells Center Director Raghu Mirmira, MD, PhD<~ ink.aspx?_id="92302C6E9AFF496F9C96F9952B50ABB7&_z=za">. “His group has the expertise to deliver genes to specific cells in a way that could fundamentally reverse—or cure—the diseases we study.”<~ ink.aspx?_id="92302C6E9AFF496F9C96F9952B50ABB7&_z=zp">

Herzog, a leader in his field, is well known by his peers for his collaboration in the study and development of oral therapies to deter inhibitor formation in patients with hemophilia–a side effect experienced by nearly one-third of those treated with clotting factor replacement therapies. In a novel approach, Herzog and his research group have collaborated with plant biotechnology experts to harvest lettuce plants that express useful proteins in their green leaves. These transgenic lettuce cells may be used to induce tolerance to current treatments. Advancements in this area would not only provide easier treatment options for children, but would be a welcomed alternative to the invasive and costly injection therapies that are used today.<~ ink.aspx?_id="92302C6E9AFF496F9C96F9952B50ABB7&_z=zp">

The Gene and Cell Therapy Program is the fourth new addition to the Wells Center in the last decade. Herzog is among only a handful of recruits to result from the Indiana Collaborative Initiative for Talent Enrichment (INCITE), an initiative funded by Lilly Endowment Inc. INCITE launched in 2017 with the mission to enhance research and education in Indiana while strengthening the state’s economic health. For IU School of Medicine, INCITE fosters growth and creates opportunity for collaboration while progressing the school’s mission to improve health in the state of Indiana and beyond.<~ ink.aspx?_id="92302C6E9AFF496F9C96F9952B50ABB7&_z=zp">

The views expressed in this content represent the perspective and opinions of the author and may or may not represent the position of Indiana University School of Medicine.

Sara Buckallew

Communications Coordinator

Sara Buckallew works in the Dean's Office of Strategic Communications. As a communications coordinator, Sara supports internal and external communication needs for the Herman B Wells Center for Pediatric Research and the Center for Diabetes and Metabolic...