Adeno-associated virus-mediated gene therapy in central nervous system genetic disorders

Gene therapy has become a promising strategy to treat the genetic disorders of central nervous system (CNS). In the last five decades the development and characterization of recombinant adeno-associated virus (rAAV) as a safe and effective vector for gene delivery has revolutionized this field. Development of rAAV with better efficacy and selectivity for the CNS has resulted in increasingly successful preclinical and clinical applications. This chapter provides an overview of AAV-mediated CNS gene therapy, including discovery and engineering of novel AAV capsids, advances in preclinical models of genetic disorders, and clinical translation of AAV technology.