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Redosing Adeno-Associated Virus Gene Therapy to the Central Nervous System.
McElroy, Abigail; Sena-Esteves, Miguel; Arjomandnejad, Motahareh; Keeler, Allison M; Gray-Edwards, Heather L.
Afiliação
  • McElroy A; Horae Gene Therapy Center and The Li Weibo Institute for Rare Diseases Research, Worcester, Massachusetts, USA.
  • Sena-Esteves M; Horae Gene Therapy Center and The Li Weibo Institute for Rare Diseases Research, Worcester, Massachusetts, USA.
  • Arjomandnejad M; Department of Neurology, Worcester, Massachusetts, USA.
  • Keeler AM; Horae Gene Therapy Center and The Li Weibo Institute for Rare Diseases Research, Worcester, Massachusetts, USA.
  • Gray-Edwards HL; Horae Gene Therapy Center and The Li Weibo Institute for Rare Diseases Research, Worcester, Massachusetts, USA.
Hum Gene Ther ; 33(17-18): 889-892, 2022 09.
Article em En | MEDLINE | ID: mdl-36074937
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.
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Dependovirus / Vetores Genéticos Tipo de estudo: Risk_factors_studies Limite: Humans Idioma: En Revista: Hum Gene Ther Assunto da revista: GENETICA MEDICA / TERAPEUTICA Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Estados Unidos

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Dependovirus / Vetores Genéticos Tipo de estudo: Risk_factors_studies Limite: Humans Idioma: En Revista: Hum Gene Ther Assunto da revista: GENETICA MEDICA / TERAPEUTICA Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Estados Unidos