A Single Multiplex crRNA Array for FnCpf1-Mediated Human Genome Editing.
Mol Ther
; 26(8): 2070-2076, 2018 08 01.
Article
em En
| MEDLINE
| ID: mdl-29910177
ABSTRACT
Cpf1 has been harnessed as a tool for genome manipulation in various species because of its simplicity and high efficiency. Our recent study demonstrated that FnCpf1 could be utilized for human genome editing with notable advantages for target sequence selection due to the flexibility of the protospacer adjacent motif (PAM) sequence. Multiplex genome editing provides a powerful tool for targeting members of multigene families, dissecting gene networks, modeling multigenic disorders in vivo, and applying gene therapy. However, there are no reports at present that show FnCpf1-mediated multiplex genome editing via a single customized CRISPR RNA (crRNA) array. In the present study, we utilize a single customized crRNA array to simultaneously target multiple genes in human cells. In addition, we also demonstrate that a single customized crRNA array to target multiple sites in one gene could be achieved. Collectively, FnCpf1, a powerful genome-editing tool for multiple genomic targets, can be harnessed for effective manipulation of the human genome.
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Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Genoma Humano
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Análise de Sequência com Séries de Oligonucleotídeos
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Endonucleases
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Edição de Genes
Limite:
Humans
Idioma:
En
Ano de publicação:
2018
Tipo de documento:
Article