Comparison of the editing patterns and editing efficiencies of TALEN and CRISPR-Cas9 when targeting the human CCR5 gene



Título del documento: Comparison of the editing patterns and editing efficiencies of TALEN and CRISPR-Cas9 when targeting the human CCR5 gene
Revue: Genetics and molecular biology
Base de datos: PERIÓDICA
Número de sistema: 000417988
ISSN: 1415-4757
Autores: 1
2
1
3
1
Instituciones: 1Universidade Federal do Rio de Janeiro, Laboratorio de Virologia Molecular, Rio de Janeiro. Brasil
2Fundacao Oswaldo Cruz, Instituto de Ciencia e Tecnologia em Biomodelos, Rio de Janeiro. Brasil
3Fundacao Oswaldo Cruz, Laboratorio de Pesquisas sobre o Timo, Rio de Janeiro. Brasil
Año:
Periodo: Mar
Volumen: 41
Número: 1
Paginación: 167-179
País: Brasil
Idioma: Inglés
Tipo de documento: Artículo
Enfoque: Experimental
Resumen en inglés The human C-C chemokine receptor type-5 (CCR5) is the major transmembrane co-receptor that mediates HIV-1 entry into target CD4+ cells. Gene therapy to knock-out the CCR5 gene has shown encouraging results in providing a functional cure for HIV-1 infection. In gene therapy strategies, the initial region of the CCR5 gene is a hotspot for producing functional gene knock-out. Such target gene editing can be done using programmable endonucleases such as transcription activator-like effector nucleases (TALEN) or clustered regularly interspaced short palindromic repeats (CRISPR-Cas9). These two gene editing approaches are the most modern and effective tools for precise gene modification. However, little is known of potential differences in the efficiencies of TALEN and CRISPR-Cas9 for editing the beginning of the CCR5 gene. To examine which of these two methods is best for gene therapy, we compared the patterns and amount of editing at the beginning of the CCR5 gene using TALEN and CRISPR-Cas9 followed by DNA sequencing. This comparison revealed that CRISPR-Cas9 mediated the sorting of cells that contained 4.8 times more gene editing than TALEN+ transfected cells
Disciplinas: Medicina,
Química,
Biología
Palabras clave: Terapéutica y rehabilitación,
Bioquímica,
Virus,
Terapia génica,
Gen CCR5,
CRISPR-Cas9,
Eficiencia,
Edición de genes,
TALEN
Keyword: Therapeutics and rehabilitation,
Biochemistry,
Virus,
Gene therapy,
CCR5 gene,
CRISPR-Cas9,
Efficiency,
Gene edition,
TALEN
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