Redosing Adeno-Associated Virus Gene Therapy to the Central Nervous System
Abigail McElroy, Miguel Sena‐Esteves, Motahareh Arjomandnejad, Allison M. Keeler, Heather Gray‐Edwards
Abstract
Adeno-associated virus (AAV)-mediated gene therapies have provided promising treatments for numerous neurological disorders. Redosing of AAV to the central nervous system (CNS) is an attractive research area due to both the somewhat immunologically privileged status of the CNS as well as the possibility of reduced glial transgene expression over time following a single injection. Continued study of the immune responses to both intraparenchymal and intra-CSF delivery of AAV mediated gene therapies, as well as the continued study of immunosuppressive regimens, could allow for eventual redosing in patients.
Topics & Concepts
Adeno-associated virusCentral nervous systemGenetic enhancementTransgeneVirusMedicineImmune systemGene deliveryImmunologyNervous systemVirologyBiologyNeuroscienceGeneVector (molecular biology)GeneticsRecombinant DNAVirus-based gene therapy researchRNA Interference and Gene DeliveryViral Infections and Immunology Research