A Compact, High-Accuracy Cas9 with a Dinucleotide PAM for In Vivo Genome Editing.

Edraki, Alireza; Mir, Aamir; Ibraheim, Raed; Gainetdinov, Ildar; Yoon, Yeonsoo; Song, Chun-Qing; Cao, Yueying; Gallant, Judith; Xue, Wen; Rivera-Pérez, Jaime A; Sontheimer, Erik J

A Compact, High-Accuracy Cas9 with a Dinucleotide PAM for In Vivo Genome Editing.

Edraki, Alireza; Mir, Aamir; Ibraheim, Raed; Gainetdinov, Ildar; Yoon, Yeonsoo; Song, Chun-Qing; Cao, Yueying; Gallant, Judith; Xue, Wen; Rivera-Pérez, Jaime A; Sontheimer, Erik J.

Afiliação

Edraki A; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA 01605, USA.
Mir A; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA 01605, USA.
Ibraheim R; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA 01605, USA.
Gainetdinov I; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA 01605, USA.
Yoon Y; Department of Pediatrics, Division of Genes and Development, University of Massachusetts Medical School, Worcester, MA 01605, USA.
Song CQ; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA 01605, USA.
Cao Y; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA 01605, USA.
Gallant J; Department of Pediatrics, Division of Genes and Development, University of Massachusetts Medical School, Worcester, MA 01605, USA.
Xue W; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA 01605, USA; Program in Molecular Medicine, University of Massachusetts Medical School, Worcester, MA 01605, USA.
Rivera-Pérez JA; Department of Pediatrics, Division of Genes and Development, University of Massachusetts Medical School, Worcester, MA 01605, USA.
Sontheimer EJ; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA 01605, USA; Program in Molecular Medicine, University of Massachusetts Medical School, Worcester, MA 01605, USA. Electronic address: erik.sontheimer@umassmed.edu.

Mol Cell ; 73(4): 714-726.e4, 2019 02 21.

Article em En | MEDLINE | ID: mdl-30581144

Assuntos

Proteína 9 Associada à CRISPR/genética; Sistemas CRISPR-Cas; Repetições Palindrômicas Curtas Agrupadas e Regularmente Espaçadas; DNA/genética; Edição de Genes/métodos; Fígado/enzimologia; Neisseria meningitidis/enzimologia; Pró-Proteína Convertase 9/genética; Animais; Proteína 9 Associada à CRISPR/metabolismo; DNA/metabolismo; Dependovirus/genética; Transferência Embrionária; Feminino; Vetores Genéticos; Células HEK293; Humanos; Células K562; Camundongos Endogâmicos C57BL; Motivos de Nucleotídeos; Pró-Proteína Convertase 9/metabolismo; RNA Guia de Cinetoplastídeos/genética; RNA Guia de Cinetoplastídeos/metabolismo; Especificidade por Substrato; Zigoto/metabolismo

Palavras-chave

CRISPR; Neisseria; Nme2Cas9; PAM-interacting domain; adeno-associated virus; anti-CRISPR; off-target; protospacer adjacent motif; sgRNA

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Texto completo: 1 Base de dados: MEDLINE Assunto principal: DNA / Repetições Palindrômicas Curtas Agrupadas e Regularmente Espaçadas / Sistemas CRISPR-Cas / Pró-Proteína Convertase 9 / Edição de Genes / Proteína 9 Associada à CRISPR / Fígado / Neisseria meningitidis Limite: Animals / Female / Humans Idioma: En Ano de publicação: 2019 Tipo de documento: Article

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