Biochemical recovery and purification of gene therapy vectors

Abstract

Rapid progress has been made with the molecular design of novel viral and non-viral gene therapy vectors. Exploiting upstream processes of producer cell-culture and downstream operations adapted from protein recovery, vectors have been accumulated in quantities and purities appropriate for the initiation of clinical trials, It is not clear, however, if such methodologies will be appropriate for efficient operation at the manufacturing scales required for clinically successful vectors. Technologies suited to the fractionation of nanoparticles may bypass practical bottlenecks experienced by current processes. The behaviour in such fractionation systems of natural and synthetic particles, which variously mimic the size, density and surface chemistry of vector products, could benefit the improved design of efficient manufacture for gene therapy vectors.