PROGENRA INC — Department of Health and Human Services SBIR Phase I: DESCRIPTION (provided by applicant): Because of their degeneracy and selective involvement
PROGENRA INC — SBIR Phase I award from Department of Health and Human Services.
- Amount
- $224,512
- Agency
- Department of Health and Human Services · National Institutes of Health
- Program / Phase
- SBIR · Phase I
- Solicitation
- PA13-234
- NAICS
- —
- Place of performance
- PA
- Period
- —
Description
DESCRIPTION (provided by applicant): Because of their degeneracy and selective involvement in most cellular pathways, ubiquitin conjugating and deconjugating enzymes are being explored as therapeutic targets for treating many major diseases, including neurodegenerative disease. The proteasome-associated deubiquitylase (DUB) USP14 plays a key role in regulating proteasomal degradation of pathogenic proteins such as tau, TDP-43 and ?-synuclein. Huntington's disease (HD) is a progressive, fatal neurodegenerative disease initially characterized by chorea and gait disturbances. The causative factor is accumulation of polyglutamine (poly Q) expanded huntingtin (Htt) protein, which is polyubiquitylated, but accumulates in inclusion bodies (IBs), rather than being degraded by the proteasome. Results from in vivo models link enhanced degradation of poly Q expanded Htt with reversal of behavioral deficits. Since USP14 has been shown to inhibit degradation of pathogenic, neurotoxic proteins, it is hypothesized that