NF1 Stem Cell Project
One of the major barriers to identifying new treatments for individuals with NF1 is a lack of human tissue specimens. To overcome this, investigators at the Washington University NF Center launched an exciting new initiative, called the NF1 Stem Cell Project (NSCP), in October 2012.
About the NSCP
The NSCP leverages new technologies in cellular engineering to generate brain cells from a skin biopsy, urine sample, or blood sample. Using this approach, Dr. Corina Anastasaki, a senior Staff Scientist in the laboratory of David H. Gutmann, MD, PhD, has established a collection of reprogrammed nerve cells for drug discovery and evaluation. The availability of these precious resources enables investigators to study the effects of impaired NF1 gene function on actual human brain cells.
Participating in the NSCP
The NSCP is recruiting individuals with specific NF1-associated features including optic pathway gliomas, malignant nerve sheath tumors and autism spectrum disorder. By understanding how changes in the NF1 gene lead to specific medical problems in NF1, researchers at the Washington University NF Center aim to develop personalized treatments of individuals with NF1.
We are deeply grateful to those individuals with NF1 who have already provided samples to the NSCP. Advances in the treatment of NF1 would not be possible without the dedication of our families living with NF1.