rAAV2.5T-Cre

rAAV2.5T-Cre is a recombinant adeno-associated virus (rAAV) gene therapy vector that utilizes the 2.5T capsid to deliver the Cre recombinase gene. It is designed for gene editing applications in the airway epithelium, particularly for the treatment of cystic fibrosis. The 2.5T capsid is characterized by its unique ability to efficiently transduce airway basal cells—the primary progenitor cells of the conducting airways—without the need for disrupting the epithelial barrier. In experimental models, such as the CFTRint1-eGFP(lsl) ferret, rAAV2.5T-Cre mediates site-specific DNA recombination to excise loxP-flanked stop cassettes, thereby enabling the reactivation of the endogenous CFTR gene or the activation of reporter genes. This vector represents a promising tool for durable, progenitor-directed gene therapy strategies in the lungs.

Other names

recombinant adeno-associated virus 2.5T-Cre