Generation of Humanized Zebrafish Models for the In Vivo Assessment of Antisense Oligonucleotide-Based Splice Modulation Therapies.

Schellens, Renske; de Vrieze, Erik; Slijkerman, Ralph; Kremer, Hannie; van Wijk, Erwin

Schellens, Renske; de Vrieze, Erik; Slijkerman, Ralph; Kremer, Hannie; van Wijk, Erwin.

Afiliación

Schellens R; Department of Otorhinolaryngology, Radboud University Medical Center, Nijmegen, The Netherlands.
de Vrieze E; Donders Institute for Brain Cognition and Behaviour, Nijmegen, The Netherlands.
Slijkerman R; Department of Otorhinolaryngology, Radboud University Medical Center, Nijmegen, The Netherlands.
Kremer H; Donders Institute for Brain Cognition and Behaviour, Nijmegen, The Netherlands.
van Wijk E; Department of Otorhinolaryngology, Radboud University Medical Center, Nijmegen, The Netherlands.

Methods Mol Biol ; 2434: 281-299, 2022.

Article en En | MEDLINE | ID: mdl-35213025

Asunto(s)

Empalme del ARN; Pez Cebra; Animales; Humanos; Mutación; Oligonucleótidos Antisentido/farmacología; Precursores del ARN/genética; Pez Cebra/genética; Pez Cebra/metabolismo

Palabras clave

Antisense oligonucleotides; Inherited retinal dystrophies; Pre-mRNA splicing; Species-specific minigene splice assay; Usher syndrome; Zebrafish

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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Pez Cebra / Empalme del ARN Tipo de estudio: Guideline Límite: Animals / Humans Idioma: En Revista: Methods Mol Biol Asunto de la revista: BIOLOGIA MOLECULAR Año: 2022 Tipo del documento: Article País de afiliación: Países Bajos

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