To CRISPR and beyond: the evolution of genome editing in stem cells
Autor: | Paul S. Knoepfler, Kuang Yui Chen |
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Rok vydání: | 2016 |
Předmět: |
Gene Editing
0301 basic medicine Genetics Embryology Transcription activator-like effector nuclease Genome Human Stem Cells Biomedical Engineering Computational biology Biology Endonucleases Genome Zinc finger nuclease Genome engineering 03 medical and health sciences 030104 developmental biology Genome editing Perspective Animals Humans CRISPR Clustered Regularly Interspaced Short Palindromic Repeats Stem cell Genetic Engineering Gene |
Zdroj: | Regenerative Medicine. 11:801-816 |
ISSN: | 1746-076X 1746-0751 |
DOI: | 10.2217/rme-2016-0107 |
Popis: | The goal of editing the genomes of stem cells to generate model organisms and cell lines for genetic and biological studies has been pursued for decades. There is also exciting potential for future clinical impact in humans. While recent, rapid advances in targeted nuclease technologies have led to unprecedented accessibility and ease of gene editing, biology has benefited from past directed gene modification via homologous recombination, gene traps and other transgenic methodologies. Here we review the history of genome editing in stem cells (including via zinc finger nucleases, transcription activator-like effector nucleases and CRISPR–Cas9), discuss recent developments leading to the implementation of stem cell gene therapies in clinical trials and consider the prospects for future advances in this rapidly evolving field. |
Databáze: | OpenAIRE |
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