Transcriptome and proteome showed shared dysregulations between replicate tumors providing putative targets to overcome resistance. In vivo CRISPR/Cas9 dropout screens in PDX revealed broad dependency on BCL2, BRIP1 and COPS2. Accordingly, venetoclax re-sensitized derivative tumors towards chemotherapy, ...
In summary, we have developed a CRISPR–Cas9-based screening approach, providing a paradigm for in vivo genetic modifier studies of other repeat expansion disorders that represent a growing class of currently intractable human diseases. This has allowed us to systematically test a large number of ge...
et al. Genome-scale in vivo CRISPR screen identifies RNLS as a target for beta cell protection in type 1 diabetes. Nat Metab 2, 934–945 (2020). https://doi.org/10.1038/s42255-020-0254-1 Download citation Received26 May 2020 Accepted26 June 2020 Published27 July 2020 Issue DateSeptember...
CRISPR–Cas9 screen to boost old NSCs To systematically identify genes that boost NSC activation as a function of age, we developed a genetic screening platform to conduct genome-wide CRISPR–Cas9 knockout screens in primary NSC cultures from young and old mice (Fig.1a; see Fig.2for in vivo...
CRISPR/Cas9-Mediated Genetic Engineering In Vivo Search Taconic's Model Generation Solutions database by application or model type: Genetically Engineered Models Publication Database Never Miss an Insight Sign up to receive the latest scientific news & insights covering all areas of preclinicalin vivore...
Genome-wide CRISPR screens reveal host factors critical for SARS-CoV-2 infection. Cell 184, 76–91 (2021). Article CAS PubMed Google Scholar Doench, J. G. et al. Rational design of highly active sgRNAs for CRISPR–Cas9-mediated gene inactivation. Nat. Biotechnol. 32, 1262–1267 (2014)...
(CRISPRa) domains with deactivated Cas9 proteins (dCas9). These tools have been utilized in genome-scale CRISPRi/a screen to recognize hereditary modifiers that are synergistic or opposing to malady mutations in an orderly and fair manner, thereby identifying illness mechanisms and discovering novel...
NTLA-2001 is an in vivo gene-editing therapeutic agent that is designed to treat ATTR amyloidosis by reducing the concentration of TTR in serum. It is based on the clustered regu-larly interspaced short palindromic repeats and associated Cas9 endonuclease (CRISPR-Cas9) system and comprises a ...
2021年6月26日,NEJM杂志发表了一篇题为:CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis 的论文,公布了诺奖得主Jennifer Doudna创立的 Intellia Therapeutics 和 再生元 合作开发治疗转甲状腺素蛋白淀粉样变性(ATTR)的CRISPR基因编辑疗法NTLA-2001I期
Table S1. In vivo CRISPR screening data, related to Figures 2 and 6 Table S2. Transcriptomic and epigenomic sequencing results, related to Figures 6 and 7 Table S3. Genes included in GSEA analysis and pathway enrichment analysis and results, related to Figure 6 Table S4. Primer sequences for...