Pharmacologic normalization of pathogenic dosage underlying genetic diseases: an overview of the literature and path forward.

Poirier, Mathieu B; Hadwen, Jeremiah; MacKenzie, Alex

Poirier, Mathieu B; Hadwen, Jeremiah; MacKenzie, Alex.

Afiliação

Poirier MB; Children's Hospital of Eastern Ontario Research Institute, Ottawa, ON, Canada K1H8L1.
Hadwen J; Children's Hospital of Eastern Ontario Research Institute, Ottawa, ON, Canada K1H8L1.
MacKenzie A; Department of Cellular and Molecular Medicine, University of Ottawa, Ottawa, ON, Canada K1H8L1.

Emerg Top Life Sci ; 3(1): 53-62, 2019 Mar 29.

Article em En | MEDLINE | ID: mdl-33523192

Palavras-chave

drug therapy; gene regulation; haploinsufficiency; rare genetic disease; rescuing paralog

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Revista: Emerg Top Life Sci Ano de publicação: 2019 Tipo de documento: Article

Texto completo

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PubMed Links

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Revista: Emerg Top Life Sci Ano de publicação: 2019 Tipo de documento: Article