This technology is a conditional human ALFY protein-expressing mouse model for the study of diseases driven by pathological protein aggregation in the nervous system.
Recent human genetic and experimental data suggest autophagy-linked FYVE protein (ALFY) overexpression as a promising disease-modifying strategy for Huntington’s disease and related proteinopathies. Although there is strong genetic evidence for its utility, there are currently no in vivo models that permit tissue-specific control of hALFY expression.
This technology is a hALFY knock-in mouse model that permits the localized study of hALFY expression via tissue-specific delivery of Cre recombinase, which excises a floxed stop cassette at the Rosa26 locus. Expression at this locus supports predictable, ubiquitous, controllable transgene expression without disrupting endogenous gene function. This model can be used to test hALFY overexpression for clearing pathological protein aggregation in the context of neurodegenerative diseases such as Huntington’s disease.
This technology has been validated in vivo using several proteinopathy models, including those for Huntington’s disease.
IR CU26143
Licensing Contact: Kristin Neuman
