Next-generation strategies for gene-targeted therapies of central nervous system disorders: A workshop summary.

Morris, Jill A; Boshoff, Chris H; Schor, Nina F; Wong, Ling M; Gao, Guangping; Davidson, Beverly L

Morris, Jill A; Boshoff, Chris H; Schor, Nina F; Wong, Ling M; Gao, Guangping; Davidson, Beverly L.

Afiliação

Morris JA; National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD 20892, USA. Electronic address: jill.morris@nih.gov.
Boshoff CH; National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD 20892, USA.
Schor NF; National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD 20892, USA.
Wong LM; National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD 20892, USA.
Gao G; Horae Gene Therapy Center, UMass Medical School, Worcester, MA 01605, USA.
Davidson BL; Children's Hospital of Philadelphia and University of Pennsylvania, Philadelphia, PA 19104, USA. Electronic address: davidsonbl@chop.edu.

Mol Ther ; 29(12): 3332-3344, 2021 12 01.

Article em En | MEDLINE | ID: mdl-34547463

Assuntos

Doenças do Sistema Nervoso Central; Doenças do Sistema Nervoso Central/genética; Doenças do Sistema Nervoso Central/terapia; Técnicas de Transferência de Genes; Terapia Genética; Humanos

Palavras-chave

AAV vectors; antisense oligonucleotides; central nervous system; gene-targeted therapies; ultra-rare diseases

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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Doenças do Sistema Nervoso Central Limite: Humans Idioma: En Ano de publicação: 2021 Tipo de documento: Article

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