| Autor: |
Fellmann, Christof, Gowen, Benjamin G, Lin, Pei-Chun, Doudna, Jennifer A, Corn, Jacob E |
| Rok vydání: |
2017 |
| Předmět: |
|
| Zdroj: |
Nature reviews. Drug discovery, vol 16, iss 2 |
| Popis: |
The recent development of CRISPR-Cas systems as easily accessible and programmable tools for genome editing and regulation is spurring a revolution in biology. Paired with the rapid expansion of reference and personalized genomic sequence information, technologies based on CRISPR-Cas are enabling nearly unlimited genetic manipulation, even in previously difficult contexts, including human cells. Although much attention has focused on the potential of CRISPR-Cas to cure Mendelian diseases, the technology also holds promise to transform the development of therapies to treat complex heritable and somatic disorders. In this Review, we discuss how CRISPR-Cas can affect the next generation of drugs by accelerating the identification and validation of high-value targets, uncovering high-confidence biomarkers and developing differentiated breakthrough therapies. We focus on the promises, pitfalls and hurdles of this revolutionary gene-editing technology, discuss key aspects of different CRISPR-Cas screening platforms and offer our perspectives on the best practices in genome engineering. |
| Databáze: |
OpenAIRE |
| Externí odkaz: |
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