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    Redosing Adeno-Associated Virus Gene Therapy to the Central Nervous System

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    Authors
    McElroy, Abigail
    Sena-Esteves, Miguel
    Arjomandnejad, Motahareh
    Keeler, Allison M
    Gray-Edwards, Heather L
    UMass Chan Affiliations
    Horae Gene Therapy Center
    Li Weibo Institute for Rare Diseases Research
    Neurology
    Pediatrics
    Radiology
    Document Type
    Journal Article
    Publication Date
    2022-09-16
    Keywords
    AAV
    central nervous system
    gene therapy
    redosing
    
    Metadata
    Show full item record
    Link to Full Text
    https://doi.org/10.1089/hum.2022.170
    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.
    Source
    McElroy 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.
    DOI
    10.1089/hum.2022.170
    Permanent Link to this Item
    http://hdl.handle.net/20.500.14038/51575
    PubMed ID
    36074937
    ae974a485f413a2113503eed53cd6c53
    10.1089/hum.2022.170
    Scopus Count
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