Efficient gene correction of an aberrant splice site in ß-thalassaemia iPSCs by CRISPR/Cas9 and single-strand oligodeoxynucleotides.

Xiong, Zeyu; Xie, Yingjun; Yang, Yi; Xue, Yanting; Wang, Ding; Lin, Shouheng; Chen, Diyu; Lu, Dian; He, Lina; Song, Bing; Yang, Yinghong; Sun, Xiaofang

Xiong, Zeyu; Xie, Yingjun; Yang, Yi; Xue, Yanting; Wang, Ding; Lin, Shouheng; Chen, Diyu; Lu, Dian; He, Lina; Song, Bing; Yang, Yinghong; Sun, Xiaofang.

Afiliação

Xiong Z; Key Laboratory for Major Obstetric Diseases of Guangdong Province, Key Laboratory of Reproduction and Genetics of Guangdong Higher Education Institutes, The Third Affiliated Hospital of Guangzhou Medical University, Guangzhou, Guangdong, China.
Xie Y; Key Laboratory for Major Obstetric Diseases of Guangdong Province, Key Laboratory of Reproduction and Genetics of Guangdong Higher Education Institutes, The Third Affiliated Hospital of Guangzhou Medical University, Guangzhou, Guangdong, China.
Yang Y; Key Laboratory for Major Obstetric Diseases of Guangdong Province, Key Laboratory of Reproduction and Genetics of Guangdong Higher Education Institutes, The Third Affiliated Hospital of Guangzhou Medical University, Guangzhou, Guangdong, China.
Xue Y; Key Laboratory for Major Obstetric Diseases of Guangdong Province, Key Laboratory of Reproduction and Genetics of Guangdong Higher Education Institutes, The Third Affiliated Hospital of Guangzhou Medical University, Guangzhou, Guangdong, China.
Wang D; Key Laboratory for Major Obstetric Diseases of Guangdong Province, Key Laboratory of Reproduction and Genetics of Guangdong Higher Education Institutes, The Third Affiliated Hospital of Guangzhou Medical University, Guangzhou, Guangdong, China.
Lin S; Key Laboratory for Major Obstetric Diseases of Guangdong Province, Key Laboratory of Reproduction and Genetics of Guangdong Higher Education Institutes, The Third Affiliated Hospital of Guangzhou Medical University, Guangzhou, Guangdong, China.
Chen D; Key Laboratory for Major Obstetric Diseases of Guangdong Province, Key Laboratory of Reproduction and Genetics of Guangdong Higher Education Institutes, The Third Affiliated Hospital of Guangzhou Medical University, Guangzhou, Guangdong, China.
Lu D; Key Laboratory for Major Obstetric Diseases of Guangdong Province, Key Laboratory of Reproduction and Genetics of Guangdong Higher Education Institutes, The Third Affiliated Hospital of Guangzhou Medical University, Guangzhou, Guangdong, China.
He L; Key Laboratory for Major Obstetric Diseases of Guangdong Province, Key Laboratory of Reproduction and Genetics of Guangdong Higher Education Institutes, The Third Affiliated Hospital of Guangzhou Medical University, Guangzhou, Guangdong, China.
Song B; Key Laboratory for Major Obstetric Diseases of Guangdong Province, Key Laboratory of Reproduction and Genetics of Guangdong Higher Education Institutes, The Third Affiliated Hospital of Guangzhou Medical University, Guangzhou, Guangdong, China.
Yang Y; Key Laboratory for Major Obstetric Diseases of Guangdong Province, Key Laboratory of Reproduction and Genetics of Guangdong Higher Education Institutes, The Third Affiliated Hospital of Guangzhou Medical University, Guangzhou, Guangdong, China.
Sun X; Key Laboratory for Major Obstetric Diseases of Guangdong Province, Key Laboratory of Reproduction and Genetics of Guangdong Higher Education Institutes, The Third Affiliated Hospital of Guangzhou Medical University, Guangzhou, Guangdong, China.

J Cell Mol Med ; 23(12): 8046-8057, 2019 12.

Article em En | MEDLINE | ID: mdl-31631510

Assuntos

Sistemas CRISPR-Cas; Edição de Genes/métodos; Células-Tronco Pluripotentes Induzidas/metabolismo; Oligodesoxirribonucleotídeos/genética; Splicing de RNA/genética; Globinas beta/genética; Talassemia beta/genética; DNA de Cadeia Simples/genética; Terapia Genética/métodos; Hematopoese; Humanos; Células-Tronco Pluripotentes Induzidas/química; Mutação; Clivagem do RNA/genética; Sítios de Splice de RNA; Sequenciamento do Exoma; Globinas beta/metabolismo; Talassemia beta/metabolismo

Palavras-chave

CRISPR/Cas9; HBB gene; gene correction; induced pluripotent stem cells; secondary cleavage; single-stranded oligodeoxynucleotide; ß-Thalassaemia

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Oligodesoxirribonucleotídeos / Splicing de RNA / Talassemia beta / Globinas beta / Células-Tronco Pluripotentes Induzidas / Sistemas CRISPR-Cas / Edição de Genes Limite: Humans Idioma: En Revista: J Cell Mol Med Ano de publicação: 2019 Tipo de documento: Article

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