Here, the authors discuss how patient-derived organoids should be grown and how advanced genome-editing tools may be applied to research on modeling of cancer and infectious diseases. They also highlight practical applications of organoids ranging from basic studies to drug screening and precision medicine.
Herein, we highlight the power of using genetic complementation approaches and CRISPR/Cas9-mediated editing for drug-target validation ex vivo and in vivo, linking the anti-tumor properties of rocaglates to eIF4A inhibition.
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In this work, the authors highlight the power of using genetic complementation approaches and CRISPR/Cas9-mediated editing for drug-target validation ex vivo and in vivo, linking the anti-tumor properties of rocaglates to eIF4A inhibition.
In this study, the authors used the CRISPR system to generate a knock-in reporter human iPS cell line for MYF5, as an early myogenic specification gene, to allow prospective identification and purification of myogenic progenitors from human iPS cells. Furthermore, in order to prove the reporter function, endogenous MYF5 expression was induced using a novel dead Cas9-VP160 transcriptional activator. These data provides valuable guidelines for generation of knock-in reporter human iPS cell lines for myogenic genes which can be used for disease modeling, drug screening, gene correction and future in vivo applications.
In this study, the scientists highlight the power of using genetic complementation approaches and CRISPR/Cas9-mediated editing for drug-target validation ex vivo and in vivo, linking the anti-tumor properties of rocaglates to eIF4A inhibition.
Resources for genome-scale loss-of-function screens in mammalian cells have progressed rapidly, particularly with RNA interference (RNAi)-based libraries and, more recently, with CRISPR–Cas9-based libraries. A new parallel screening study leverages the complementary strengths of each system to dissect antiviral drug mechanisms and to identify potential combination therapy strategies.
In this study, the scientists used the CRISPR-Cas9 system to target exons encoding functional protein domains. A screen of 192 chromatin regulatory domains in murine acute myeloid leukemia cells identifies six known drug targets and 19 additional dependencies.
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Here, the authors discuss how patient-derived organoids should be grown and how advanced genome-editing tools may be applied to research on modeling of cancer and infectious diseases. They also highlight practical applications of organoids ranging from basic studies to drug screening and precision medicine.