Mechanism and evidence of nonsense suppression therapy for genetic eye disorders.

Richardson, Rose; Smart, Matthew; Tracey-White, Dhani; Webster, Andrew R; Moosajee, Mariya

Richardson, Rose; Smart, Matthew; Tracey-White, Dhani; Webster, Andrew R; Moosajee, Mariya.

Afiliação

Richardson R; Department of Ocular Biology and Therapeutics, UCL Institute of Ophthalmology, London, UK.
Smart M; Department of Ocular Biology and Therapeutics, UCL Institute of Ophthalmology, London, UK.
Tracey-White D; Department of Ocular Biology and Therapeutics, UCL Institute of Ophthalmology, London, UK.
Webster AR; Department of Ocular Biology and Therapeutics, UCL Institute of Ophthalmology, London, UK; Moorfields Eye Hospital NHS Foundation Trust, London, UK.
Moosajee M; Department of Ocular Biology and Therapeutics, UCL Institute of Ophthalmology, London, UK; Moorfields Eye Hospital NHS Foundation Trust, London, UK. Electronic address: m.moosajee@ucl.ac.uk.

Exp Eye Res ; 155: 24-37, 2017 02.

Article em En | MEDLINE | ID: mdl-28065590

Assuntos

Códon sem Sentido; Oftalmopatias/terapia; Proteínas do Olho/genética; Terapia Genética/métodos; Oftalmopatias/genética; Oftalmopatias/metabolismo; Proteínas do Olho/metabolismo; Humanos

Palavras-chave

Aminoglycosides; Ataluren; Genetic eye disease; Nonsense mutation; Nonsense suppression therapy; Premature termination codon; Readthrough; Translational bypass

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Terapia Genética / Códon sem Sentido / Oftalmopatias / Proteínas do Olho Limite: Humans Idioma: En Revista: Exp Eye Res Ano de publicação: 2017 Tipo de documento: Article País de afiliação: Reino Unido

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