Direct gene therapy for repair of the spinal cord

For regrowth of injured nerve fibers following spinal cord injury (SCI), the environment must be favorable for axonal growth. The delivery of a therapeutic gene, beneficial for axonal growth, into the central nervous system for repair can be accomplished in many ways. Perhaps the most simple and elegant strategy is the so-called direct gene therapy approach that uses a single injection for delivery of a gene therapy vehicle. Among the vectors that have been used to transduce neural tissue in vivo are non-viral, herpes simplex viral, adeno-associated viral, adenoviral, and lentiviral vectors, each with their own merits and limitations. Many studies have been undertaken using direct gene therapy, ranging from strategies for neuroprotection to axonal growth promotion at the injury site, dorsal root injury repair, and initiation of a growth-supporting genetic program. The limitations and successes of direct gene transfer for spinal cord repair are discussed in this review.