CAG-targeted brain-permeable therapy tested in biallelic humanized . . . We designed a series of CAG-directed short hairpin RNAs (shRNAs) based on a previous A2 reagent, allele selective in vitro Humanized HD (Hu 128Q 21Q) and SCA3 (Ki 150Q 21Q) mice with mutant ∼100 CAGs and normal 21 CAGs alleles were used to simulate biallelic conditions occurring in patients
CAG-targeted brain-permeable therapy tested in biallelic humanized . . . We designed a series of CAG-directed short hairpin RNAs (shRNAs) based on a previous A2 reagent, allele-selective in vitro Humanized HD (Hu 128Q 21Q) and SCA3 (Ki 150Q 21Q) mice with mutant ∼100 CAGs and normal 21 CAGs alleles were used to simulate biallelic conditions occurring in patients
CAG-targeted brain-permeable therapy tested in biallelic humanized . . . We designed a series of CAG-directed short hairpin RNAs (shRNAs) based on a pre-vious A2 reagent, allele selective in vitro Humanized HD (Hu128Q 21Q) and SCA3 (Ki150Q 21Q) mice with mutant 100 CAGs and normal 21 CAGs alleles were used to simulate bial-lelic conditions occurring in patients
CAG-targeted brain-permeable therapy tested in biallelic humanized . . . We demonstrate that optimized CAG-targeted A4 (P10) and A4 (P10,11) shReagents can lower mutant huntingtin and ataxin-3 protein and its aggregates by targeting brain regions selectively and with diminished toxicity compared to other tested shRNAs
CAG-targeted brain-permeable therapy tested in biallelic humanized . . . This study demonstrates that optimized CAG-targeted shRNA reagents effectively lower toxic proteins in polyglutamine (polyQ) diseases like Huntington disease (HD) and spinocerebellar ataxia type 3 (SCA3) in vivo, paving the way for new therapies
AAV-Mediated CAG-Targeting Selectively Reduces Polyglutamine . . . - MDPI The aim of the current study was to develop and validate a therapeutic approach applicable to CAG polyQ SCAs using a CAG repeat-targeting strategy to lower the expression of expanded-polyQ proteins in vulnerable cerebellar neurons