AAV1-Δ27-264 CFTR

AAV1-Δ27-264 CFTR is an **experimental gene therapy** vector comprising an adeno-associated virus serotype 1 (AAV1) that delivers a truncated form of the cystic fibrosis transmembrane conductance regulator (CFTR) gene, specifically deleting amino acids 27-264. The construct is designed to restore CFTR function where it is deficient due to genetic mutation, and is packaged with regulatory sequences to drive gene expression in target cells. The mechanism involves direct gene supplementation: the vector infects target cells, persisting episomally, leading to transcription and translation of the truncated CFTR, which can functionally complement loss-of-function CFTR typically in cystic disease settings. The primary therapeutic investigational focus is **autosomal recessive polycystic kidney disease (ARPKD)** and, experimentally, **autosomal dominant polycystic kidney disease (ADPKD)**, with prior research context in cystic fibrosis airway rescue. The vector is notably under study by Johns Hopkins University researchers for gene delivery targeting cystic epithelia in the liver and kidney[2][3][4].

Other names

truncated CFTR AAV1 vectorΔ27-264 CFTR AAV1AAV1-truncated CFTRAAV-1-truncated CFTRAAV 1-truncated CFTR