A team led by Johns Hopkins and Kennedy Kreiger Institute researchers has developed a new gene therapy strategy that may overcome longstanding barriers to treating neurofibromatosis type 1 (NF1) as well as other conditions related to a single gene alteration.  The experimental therapy combines a miniaturized version of the NF1 gene with a uniquely engineered adeno-associated virus (AAV) vector that efficiently targets tumor tissue while limiting uptake in the liver and other healthy organs. This is an important development, as nontargeted uptake is a significant limitation of naturally occurring (nonengineered) AAV products.

In an exciting development for the neurofibromatosis (NF) community, Project Dream, an initiative launched by NF Network in 2023, has made strides in funding innovative research focused on high-potential NF projects. One of the awarded researchers, Dr. Angela Hirbe, an Associate Professor of Medicine and Pediatrics and the Director of the Adult Neurofibromatosis Clinical Program at Washington University School of Medicine, is set to present her groundbreaking research in a free virtual meeting scheduled for December 17.

Families from across Florida gathered at the Biltmore Tennis Center on November 15, for a vibrant social event that brought together learning, wellness, and play for all ages. The gathering combined useful NF education with the fun and inclusivity of the world’s fastest-growing pastime: pickleball.

Drew Sinclair, an equipment manager for the Washington Commanders, seized the opportunity on Sunday, November 30, to raise awareness for a cause close to his heart: neurofibromatosis (NF). Diagnosed with NF Type 1 at a young age, Drew is determined to use his position with the NFL to spotlight this genetic condition, which affects members of his family and 4 million people worldwide.