Gene therapy for spinal cord injury

Abstract

New insights into the pathophysiological mechanisms that contribute to the limited functional recovery after spinal cord injury (SCI) have led to the development of several novel experimental approaches. Gene therapy is one means to identify and modulate molecular mechanisms that promote plasticity and regeneration in the injured spinal cord. Therapeutic targets that can be pursued by gene delivery include the intrinsic regenerative capacity of injured neurons, the rearrangement of spared circuitry, and extrinsic factors in and around the lesion site limiting axonal regeneration. An increasing number of animal studies have also employed gene therapy in combinatorial treatments to tackle the complexity of injury-induced changes. However, several challenges regarding the effi ciency, safety, and regulation of gene expression remain to be addressed. This chapter aims to summarize different strategies for gene transfer after SCI and their translational potential.