AAV as a gene transfer vector for the treatment of neurological disorders: novel treatment thoughts for ALS.

Therapeutic delivery to the central nervous system has challenged scientists and clinicians due to the difficulty in delivering molecules and genes in an efficient manner across the blood brain barrier (BBB). This has particularly hampered efforts to deliver therapeutics to widely dispersed neurons that perish in diseases such as Amyotrophic Lateral Sclerosis (ALS), a disease affecting motor neurons throughout the brainstem and the entire spinal cord. Gene therapy has offered several potential routes to overcome the difficulties in delivering therapeutics to the brain and spinal cord. Adeno-associated viral vectors (AAV) have taken center stage for gene delivery to the central nervous system, given their ability to express genes in post mitotic cells for long periods with minimal to no toxicity. This review will focus on recent approaches to treat motor neuron disease, in particular ALS using AAV vectors.