Redosing Adeno-Associated Virus Gene Therapy to the Central Nervous System
Keeler, Allison M
Gray-Edwards, Heather L
UMass Chan AffiliationsHorae Gene Therapy Center
Li Weibo Institute for Rare Diseases Research
Document TypeJournal Article
MetadataShow full item record
AbstractAdeno-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.
SourceMcElroy A, Sena-Esteves M, Arjomandnejad M, Keeler AM, Gray-Edwards HL. Redosing Adeno-Associated Virus Gene Therapy to the Central Nervous System. Hum Gene Ther. 2022 Sep;33(17-18):889-892. doi: 10.1089/hum.2022.170. PMID: 36074937.
Permanent Link to this Itemhttp://hdl.handle.net/20.500.14038/51575