Abstract

The development and deployment of Neurofibromatosis type 1 (Nf1) mouse models provides unique opportunities to define the underlying pathogenic causes of specific NF1-associated clinical features, identify potential risk factors for their appearance and progression, and discover new therapies targeted to the responsible cellular and molecular vulnerabilities. Herein, we review the available mouse models for the common medical conditions affecting children and adults with NF1 and discuss the challenges and future directions for their use relevant to improving the care of individuals with this rare neurogenetic syndrome.

Original languageEnglish
Title of host publicationThe RASopathies
Subtitle of host publicationGenetic Syndromes of the RAS/MAPK Pathway
PublisherSpringer Nature
Pages833-854
Number of pages22
ISBN (Electronic)9783031629457
ISBN (Print)9783031629440
DOIs
StatePublished - Jan 1 2024

Keywords

  • Malignant peripheral nerve sheath tumor
  • Neurofibroma
  • Neurofibromin
  • Optic pathway glioma
  • Plexiform neurofibroma
  • RAS

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