Comprehensive characterization and validation of the Prp-hPFN1G118V mouse model: Guidelines for preclinical therapeutic testing for ALS

Comprehensive characterization and validation of the Prp-hPFN1G118V mouse model: Guidelines for preclinical therapeutic testing for ALS

The hPFN1G118V mouse model, overexpressing mutant human profilin1 linked to a rare form of ALS, was comprehensively characterized to assess its suitability for preclinical drug testing. Using a large cohort of nearly 250 transgenic and wild-type mice in a longitudinal study, we combined behavioral,...

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Bibliographic Details
Main Authors: Kaly A. Mueller, Emma G. Suneby, Matthew H. Ferola, Andy J. Moreno, Joshua D. Kidd, Kenneth Thompson, Fernando G. Vieira, Gregorio Valdez, Theo Hatzipetros
Format: Article
Language:English
Published: Elsevier 2025-08-01
Series:Neurobiology of Disease
Subjects:
Amyotrophic lateral sclerosis
Profilin1
Motor neurons
Neurofilament light chain
CMAP
Neuroinflammation
Online Access:http://www.sciencedirect.com/science/article/pii/S0969996125001913
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