AAV-dCasMINI-miniVPR

AAV-dCasMINI-miniVPR is a CRISPR-based epigenome editing gene therapy designed for the treatment of CDKL5 deficiency disorder (CDD). The therapy utilizes a compact CRISPR-associated protein, dCasMINI (a catalytically inactive or dead Cas protein), fused to the miniVPR transactivator. Delivered via a single adeno-associated virus (AAV) vector, the system is engineered to target and upregulate the expression of the CDKL2 (cyclin-dependent kinase-like 2) gene. CDKL2 is a kinase that shares functional redundancy and overlapping signaling roles with CDKL5. By increasing CDKL2 levels, the therapy aims to compensate for the loss of CDKL5 function, restore downstream phosphorylation of substrates such as EB2, and potentially rescue the motor and cognitive dysfunctions associated with CDD.

Other names

miniVPR-dCasMINI epigenome editordCasMINI-miniVPR