recombinant adeno-associated virus serotype 9 nuclear jdp

Recombinant adeno-associated virus serotype 9 nuclear JDP (rAAV9-nuclear-JDP) is an experimental gene therapy construct developed by researchers at the University of Texas Southwestern Medical Center. It utilizes an adeno-associated virus serotype 9 (AAV9) vector to deliver a J-domain protein (JDP) specifically engineered for nuclear localization. In the context of neurodegenerative research, particularly for tauopathies like Alzheimer's disease, this construct serves as a critical negative control. JDPs are molecular co-chaperones that assist Hsp70 in protein quality control, including folding, disaggregation, and degradation. While cytoplasmic JDPs have demonstrated the ability to reduce the seeding and aggregation of misfolded tau protein in the cytoplasm, the nuclear-localized version does not impact these cytoplasmic tau species, thereby validating the compartment-specific therapeutic hypothesis. It has been evaluated in the PS19 mouse model of tauopathy via hippocampal injection.

Other names

nuclear located JDP controlrAAV9/nuclear JDP