Mutant huntingtin
Biomarker1 paper1 finding
- Animals1
Interventions
1Huntingtin aggregate-responsive autophagy gene circuit
downin mice1
1 study
Huntingtin aggregate-responsive autophagy gene circuit
downin mice1
Huntingtin aggregate-responsive autophagy gene circuit decreases mutant huntingtin in mice.
“In the R6/2 HD mouse model, ARAA treatment significantly reduces mHTT burden, attenuates neuroinflammation, and rescues synaptic deficits.”
Animalsin R6/2 HD mouse modelplasmid encapsulated in CD98-targeted immunoliposomes
Synthetic gene circuit clears protein aggregates and extends lifespan in Huntington disease mice · Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 27 Sep 2026
Latest
1Animals
Synthetic gene circuit clears protein aggregates and extends lifespan in Huntington disease mice
The sensor-driven system selectively triggers autophagy regulator TFEB upon detecting toxic huntingtin conformers, lowering brain inflammation and rescuing synaptic deficits.