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 language | English |
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Title of host publication | The RASopathies |
Subtitle of host publication | Genetic Syndromes of the RAS/MAPK Pathway |
Publisher | Springer Nature |
Pages | 833-854 |
Number of pages | 22 |
ISBN (Electronic) | 9783031629457 |
ISBN (Print) | 9783031629440 |
DOIs | |
State | Published - Jan 1 2024 |
Keywords
- Malignant peripheral nerve sheath tumor
- Neurofibroma
- Neurofibromin
- Optic pathway glioma
- Plexiform neurofibroma
- RAS