[Neurofibromatosis 1: pathogenesis and therapeutic strategies--a systematic review]

Neurofibromatosis 1 (NF1, von Recklinghausen's disease) is a dominantly inherited multi-organ disease defined primarily by café au lait patches and neurofibromas. NF1 predisposes to cancer and is associated with cognitive dysfunction and learning defects. In recent years, considerable progress has been made in the understanding of NF1 pathogenesis, not least based on studies of genetically engineered animal models. We present an overview of the most important recent findings and the related current efforts to develop novel therapeutic strategies.