Increased prime edit rates in KCNQ2 and SCN1A via single nicking all-in-one plasmids.

Dirkx, N; Weuring, Wout J; De Vriendt, E; Smal, N; van de Vondervoort, J; van 't Slot, Ruben; Koetsier, M; Zonnekein, N; De Pooter, Tim; Weckhuysen, S; Koeleman, B P C

Dirkx, N; Weuring, Wout J; De Vriendt, E; Smal, N; van de Vondervoort, J; van 't Slot, Ruben; Koetsier, M; Zonnekein, N; De Pooter, Tim; Weckhuysen, S; Koeleman, B P C.

Afiliação

Dirkx N; Applied & Translational Neurogenomics Group, VIB Center for Molecular Neurology, VIB, Antwerp, Belgium. nina.dirkx@uantwerpen.vib.be.
Weuring WJ; Department of Biomedical Sciences, University of Antwerp, Antwerp, Belgium. nina.dirkx@uantwerpen.vib.be.
De Vriendt E; Department of Genetics, University Medical Center Utrecht, Utrecht, 3584 CX, The Netherlands. woutweuring2@gmail.com.
Smal N; Applied & Translational Neurogenomics Group, VIB Center for Molecular Neurology, VIB, Antwerp, Belgium.
van de Vondervoort J; Applied & Translational Neurogenomics Group, VIB Center for Molecular Neurology, VIB, Antwerp, Belgium.
van 't Slot R; Department of Biomedical Sciences, University of Antwerp, Antwerp, Belgium.
Koetsier M; Department of Genetics, University Medical Center Utrecht, Utrecht, 3584 CX, The Netherlands.
Zonnekein N; Department of Genetics, University Medical Center Utrecht, Utrecht, 3584 CX, The Netherlands.
De Pooter T; Department of Genetics, University Medical Center Utrecht, Utrecht, 3584 CX, The Netherlands.
Weckhuysen S; Applied & Translational Neurogenomics Group, VIB Center for Molecular Neurology, VIB, Antwerp, Belgium.
Koeleman BPC; Department of Biomedical Sciences, University of Antwerp, Antwerp, Belgium.

BMC Biol ; 21(1): 156, 2023 07 13.

Article em En | MEDLINE | ID: mdl-37443005

Assuntos

Sistemas CRISPR-Cas; Pequeno RNA não Traduzido; Animais; Humanos; Células HEK293; Fator 1 de Elongação de Peptídeos/genética; Plasmídeos/genética; Canal de Potássio KCNQ2/genética; Canal de Sódio Disparado por Voltagem NAV1.1/genética

Palavras-chave

CRISPR; Developmental and epileptic encephalopathy; EF-1alfa; EIEE; Gene editing; Human-induced pluripotent stem cells; KCNQ2; Monogenic diseases; Prime editing; SCN1A

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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Pequeno RNA não Traduzido / Sistemas CRISPR-Cas Idioma: En Ano de publicação: 2023 Tipo de documento: Article

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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Pequeno RNA não Traduzido / Sistemas CRISPR-Cas Idioma: En Ano de publicação: 2023 Tipo de documento: Article