AAV-HDAC5-3SA

AAV-HDAC5-3SA is an experimental gene therapy construct consisting of an adeno-associated virus (AAV) vector designed to overexpress a constitutively active mutant form of histone deacetylase 5 (HDAC5). The 3SA designation refers to the mutation of three key serine residues (S259, S280, and S498) to alanine, which prevents phosphorylation-dependent nuclear export, thereby keeping the HDAC5 protein localized in the nucleus where it can exert its epigenetic regulatory functions. In preclinical models of opioid use disorder, delivery of this construct to the nucleus accumbens (NAc) has been shown to suppress heroin-seeking behavior and relapse by regulating ion channel gene expression and reducing the firing rates of medium spiny neurons (MSNs).

Other names

AAV-HDAC5(3SA)AAV-HDAC5-S259/280/498A