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WDFY1-shRNA nanoparticles
- Development stage
- Preclinical
- Lead developer
- University of Cincinnati
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics, Nanoparticles → Drug Delivery Systems
01
Overview
WDFY1-shRNA nanoparticles is a preclinical-stage nanoparticle-encapsulated short hairpin RNA (shRNA) therapy designed to target and knock down the expression of WD repeat and FYVE domain-containing protein 1 (WDFY1). WDFY1 is an adaptor protein involved in endocytic/vacuolar membrane trafficking and inflammatory pathways, particularly in Toll-like receptor (TLR) pathways and follicular dendritic cell (FDC) antigen presentation. Developed by researchers at the University of Cincinnati College of Medicine, this therapeutic candidate is being investigated for the treatment of lupus nephritis (LN) and systemic lupus erythematosus (SLE). In preclinical mouse models of lupus nephritis, treatment with WDFY1-shRNA nanoparticles significantly improved kidney function, reduced proteinuria and blood urea nitrogen (BUN) levels, and decreased immune cell infiltration and complement C3 deposition in the kidneys.
- Other names
- nanoparticle encapsuled WDFY1-shRNAnanoparticle-encapsulated WDFY1-shRNAnanoparticle-mediated WDFY1-shRNA
02
Targets
WDFY1 (WD repeat and FYVE domain-containing protein 1)
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