Gene transfer to mouse kidney in vivo

Abstract

Genetic nephropathies represent a challenging class of disorders to be treated by gene therapy. This is primarily due to the filtering properties of the kidney itself, which does not allow the vehicle carrying the transgene of interest to remain long enough in the organ to penetrate efficiently into the nephrotic cells. Also, the kidney has a complex anatomical structure composed of different cell types compartmentalized within isolated anatomic structures that limit their access. Here, we describe a simple surgical procedure to deliver recombinant adeno-associated virus (rAAV) to the whole kidney based on the hydraulic force of the retrograde renal vein injection. In its clinical form, this procedure would correspond to a renal venography where a catheter is threaded retrograde from the femoral vein under fluoroscopic guidance.