Thursday, April 10, 2014
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Mouse model of amyotrophic
lateral sclerosis (ALS) caused by partial loss of TAR DNA
binding protein 43 (Tdp-43;
Mice with partial loss of
Tdp-43 function could be useful as models to study and screen for new
compounds to treat ALS. TDP-43 aggregation is associated with ALS. Mice were
engineered to express artificial microRNA to knock down expression of Tdp-43.
The engineered mice recapitulated ALS-like pathophysiology including
progressive neuromuscular impairments that eventually led to paralysis and
death, and neurodegeneration in the forebrain and spinal cord. Next steps
could include using the mouse model to evaluate therapeutic candidates for
Published online April 10, 2014
Patent and licensing status
Yang, C. et al. Proc.
Natl. Acad. Sci. USA; published online
March 10, 2014;
Contact: Zuoshang Xu, University of Massachusetts Medical
School, Worcester, Mass.
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