AAV as a Gene Transfer Vector for the Treatment of Neurological Disorders: Novel Treatment Thoughts for ALS
Mark E. Hester, Kevin D. Foust, Rita W. Kaspar and Brian K. Kaspar
Affiliation: Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Department of Pediatrics, The Ohio State University, 700 Children's Drive WA 3022, Columbus, OH 43205, USA.
Keywords: Gene therapy, motor neuron disease, Amyotrophic Lateral Sclerosis (ALS), Adeno-associated virus (AAV), blood brain barrier (BBB)
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. Adenoassociated 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.
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