allele-specific shRNA targeting PrP 116V

Allele-specific shRNA targeting PrP 116V is an experimental gene therapy designed to selectively suppress the expression of the mutated prion protein (PrP) allele carrying the 116V mutation (the mouse homolog of the human A117V mutation), while sparing the wild-type allele. Developed by researchers at the University of Chicago, this short-hairpin RNA (shRNA) is delivered via viral vectors (such as lentivirus or adeno-associated virus) to target the genetic variant of the PRNP gene that causes Gerstmann-Sträussler-Scheinker (GSS) disease. By selectively knocking down the mutant PrP mRNA, the therapy aims to reduce pathogenic misfolded prion protein accumulation and amyloid plaque deposition in the brain, potentially slowing the progression of GSS and other autosomal-dominant prion disorders.

Other names

allele-specific RNAi targeting PrP 116Vallele-specific shRNA targeting PrP116V