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Children’s Tumor Foundation awards $1.7 million grant to Sanford Research to study rare genetic disorder

Dr. Jill Weimer will lead team developing new pig model for Neurofibromatosis type 1

SIOUX FALLS, S.D. – A Sanford Research scientist is using an innovative pig model to better understand a rare genetic disorder that causes tumors in the nervous system, thanks to a more than $1.7 million grant from the Children’s Tumor Foundation (CTF). Jill Weimer, Ph.D., and her team received the award as part of CTF’s Synodos for NF 1 program, a unique collaboration of world-class scientists and clinicians from diverse expertise areas who work together with patients and share information in real time resulting in a faster and more efficient drug discovery and development process.

The Children’s Tumor Foundation is a not-for-profit organization dedicated to finding effective treatments for the millions of people worldwide living with neurofibromatosis (NF).

Weimer is a scientist and director of the Children’s Health Research Center at Sanford Research who studies nervous system development, neural development disorders and neurodegenerative diseases. She will lead a collaborative team of researchers from the University of Iowa, the University of Arizona and Exemplar Genetics, an Iowa-based biotechnology company that specializes in porcine models. This collaborative research crew includes experts in genetics, neuroscience, pathology medicinal chemistry, molecular biology and biomedical engineering. The group has already demonstrated success creating pig models for diseases like cystic fibrosis and ataxia telangiectasia.

“Scientists have experienced limited success replicating NF1 in mouse models; there are some characteristics of the disorder that have been difficult to model,” said Weimer. “It is becoming vital as we move toward developing effective therapies for NF1 that we develop reliable large animal models.”

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