Understanding Attention and Learning in NF1

Defining the role of the NF1 gene in normal brain development

The observation that children with NF1 have learning, memory and behavioral problems suggests that the Nf1 gene neurofibromin is important in brain development and function.

To better understand the role of neurofibromin in normal brain development, Dr. Gutmann and his colleagues are using novel strains of genetically-engineered mice, as well as human induced pluripotent stem cells (hiPSCs). These studies are aimed at determining the mechanism by which neurofibromin regulates the growth of progenitor cells (neural stem cells) and their differentiation into neurons, astrocytes and oligodendrocytes. The goal of these studies is to determine exactly how neurofibromin regulates normal brain development and function with an eye towards future treatments that leverage these new insights.

Developing new treatments for learning, behavioral, motor and sleep deficits in children with NF1

Many children with NF1 have learning difficulties, behavioral problems (autism, attention deficit), motor delays, and sleep disturbances. In an effort to develop new treatments for these common problems, cross-disciplinary studies at the Washington University NF Center are leveraging Nf1 genetically-engineered mice to complement human clinical studies led by Dr. Stephanie Morris and her colleagues in children with NF1.