CRISPR-SKIP: programmable gene splicing with single base editors
| Autor: | Nikhil Shiva, Wendy S. Woods, Pablo Perez-Pinera, Alan Luu, Jun S. Song, Michael Gapinske, Kurt A. Kostan, Jackson Winter |
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| Rok vydání: | 2018 |
| Předmět: |
0301 basic medicine
lcsh:QH426-470 Base pair Method RELA Computational biology Biology Cell Line Base editing 03 medical and health sciences Synthetic biology 0302 clinical medicine Genome editing Consensus Sequence Humans CRISPR Clustered Regularly Interspaced Short Palindromic Repeats Gene isoform lcsh:QH301-705.5 Base Pairing Gene Gene Editing Genome Base Sequence Alternative splicing High-Throughput Nucleotide Sequencing Exons PIK3CA Cytidine deaminase BRCA2 Exon skipping lcsh:Genetics 030104 developmental biology lcsh:Biology (General) RNA Splice Sites CRISPR-Cas9 030217 neurology & neurosurgery |
| Zdroj: | Genome Biology Genome Biology, Vol 19, Iss 1, Pp 1-11 (2018) |
| ISSN: | 1474-760X |
| DOI: | 10.1186/s13059-018-1482-5 |
| Popis: | CRISPR gene editing has revolutionized biomedicine and biotechnology by providing a simple means to engineer genes through targeted double-strand breaks in the genomic DNA of living cells. However, given the stochasticity of cellular DNA repair mechanisms and the potential for off-target mutations, technologies capable of introducing targeted changes with increased precision, such as single-base editors, are preferred. We present a versatile method termed CRISPR-SKIP that utilizes cytidine deaminase single-base editors to program exon skipping by mutating target DNA bases within splice acceptor sites. Given its simplicity and precision, CRISPR-SKIP will be broadly applicable in gene therapy and synthetic biology. Electronic supplementary material The online version of this article (10.1186/s13059-018-1482-5) contains supplementary material, which is available to authorized users. |
| Databáze: | OpenAIRE |
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