Genome Editing in Drug Discovery. Группа авторов
Чтение книги онлайн.
Читать онлайн книгу Genome Editing in Drug Discovery - Группа авторов страница 13
Название: Genome Editing in Drug Discovery
Автор: Группа авторов
Издательство: John Wiley & Sons Limited
Жанр: Биология
isbn: 9781119671398
isbn:
Last and perhaps one of the most exciting applications of CRISPR in drug discovery is the potential to create highly sensitive, inexpensive, point‐of‐care diagnostics for the early detection of disease (Chen et al. 2018; Gootenberg et al. 2018; Myhrvold et al. 2018). It is widely accepted, particularly in Oncology, that the probability of patient survival from the disease increases with early disease detection. The creation of diagnostics that detect cancer in stage 1 rather than when symptomatic in stage 3 or 4 will transform our ability to treat and perhaps cure this disease. Two methods have been published, described as SHERLOCK and DETECTR, that offer the potential to create such sensitive DNA diagnostics. While in early development, the potential of these innovations is huge and are being applied more broadly, including for the creation of a diagnostic test for the SARS‐CoV2 virus.
1.5 Concluding Comments
Since the demonstration of the ability of CRISPR/Cas systems to precisely and efficiently edit gene sequences in 2012, CRISPR has become embedded as a routine technique in molecular and cell biology laboratories across the field. New industries have been created to supply CRISPR reagents and CRISPR‐edited cell and animal models to the research scientist, to develop CRISPR medicines and to create CRISPR diagnostics. The applications and impact of CRISPR in drug discovery are discussed at length within this book. Within eight short years, CRISPR has transformed our ability to identify and characterize the role of new drug targets in disease and to create the cell and animal models integral to identify and optimize drug candidates. With the rate of innovation in this field, we can look forward to the development of novel CRISPR systems that increase the efficiency and specificity of gene editing, to the development of transformative CRISPR therapies with the potential to cure severe genetic diseases and to the invention of highly sensitive diagnostics for the early identification and subsequent cure of many common diseases. As we move through the coming decades, the opportunity for CRISPR to improve human health remains enormous.
References
1 Acharya, S., Mishra, A., Paul, D. et al. (2019). Francisella novicida Cas9 interrogates genomic DNA with very high specificity and can be used for mammalian genome editing. Proc. Natl. Acad. Sci. U. S. A. 116: 20959–20968.
2 Anzalone, A.V., Randolph, P.B., Davis, J.R. et al. (2019). Search‐and‐replace genome editing without double‐strand breaks or donor DNA. Nature 576: 149–157.
3 Batta, A., Kalra, B.S., and Khirasaria, R. (2020). Trends in FDA drug approvals over last 2 decades: an observational study. J Family Med Prim Care 9: 105–114.
4 Behan, F.M., Iorio, F., Picco, G. et al. (2019). Prioritization of cancer therapeutic targets using CRISPR‐Cas9 screens. Nature 568: 511–516.
5 Bibikova, M., Carroll, D., Segal, D.J. et al. (2001). Stimulation of homologous recombination through targeted cleavage by chimeric nucleases. Mol. Cell Biol. 21: 289–297.
6 Boch, J., Scholze, H., Schornack, S. et al. (2009). Breaking the code of DNA binding specificity of TAL‐type III effectors. Science 326: 1509–1512.
7 Chen, J.S., Ma, E., Harrington, L.B. et al. (2018). CRISPR‐Cas12a target binding unleashes indiscriminate single‐stranded DNase activity. Science 360: 436–439.
8 Cong, L., Ran, F.A., Cox, D. et al. (2013). Multiplex genome engineering using CRISPR/Cas systems. Science 339: 819–823.
9 Cui, Y., Cheng, X., Chen, Q. et al. (2021). CRISP‐view: a database of functional genetic screens spanning multiple phenotypes. Nucleic. Acids. Res. 49 (D1): D848–D854.
10 Doench, J.G. (2018). Am I ready for CRISPR? A user's guide to genetic screens. Nat. Rev. Genet. 19: 67–80.
11 Fellmann, C., Gowen, B.G., Lin, P.C. et al. (2017). Cornerstones of CRISPR‐Cas in drug discovery and therapy. Nat. Rev. Drug Discov. 16: 89–100.
12 Gilbert, L.A., Horlbeck, M.A., Adamson, B. et al. (2014). Genome‐scale CRISPR‐mediated control of gene repression and activation. Cell 159: 647–661.
13 Gootenberg, J.S., Abudayyeh, O.O., Kellner, M.J. et al. (2018). Multiplexed and portable nucleic acid detection platform with Cas13, Cas12a, and Csm6. Science 360: 439–444.
14 Jinek, M., Chylinski, K., Fonfara, I. et al. (2012). A programmable dual‐RNA‐guided DNA endonuclease in adaptive bacterial immunity. Science 337: 816–821.
15 Kampmann, M. (2018). CRISPRi and CRISPRa Screens in Mammalian Cells for Precision Biology and Medicine. ACS Chem. Biol. 13: 406–416.
16 Kim, Y.G., Cha, J., and Chandrasegaran, S. (1996). Hybrid restriction enzymes: zinc finger fusions to Fok I cleavage domain. Proc. Natl. Acad. Sci. U. S. A. 93: 1156–1160.
17 Lancaster, M.A., Renner, M., Martin, C.A. et al. (2013). Cerebral organoids model human brain development and microcephaly. Nature 501: 373–379.
18 Liu, X., Zhang, Y., Cheng, C. et al. (2017). CRISPR‐Cas9‐mediated multiplex gene editing in CAR‐T cells. Cell Res. 27: 154–157.
19 Lundin, A., Porritt, M.J., Jaiswal, H. et al. (2020). Development of an ObLiGaRe Doxycycline Inducible Cas9 system for pre‐clinical cancer drug discovery. Nat. Commun. 11: 4903.
20 Mali, P., Yang, L., Esvelt, K.M. et al. (2013). RNA‐guided human genome engineering via Cas9. Science 339: 823–826.
21 Morgan, P., Brown, D.G., Lennard, S. et al. (2018). Impact of a five‐dimensional framework on R&D productivity at AstraZeneca. Nat. Rev. Drug Discov. 17: 167–181.
22 Moscou, M.J. and Bogdanove, A.J. (2009). A simple cipher governs DNA recognition by TAL effectors. Science 326: 1501.
23 Myhrvold, C., Freije, C.A., Gootenberg, J.S. et al. (2018). Field‐deployable viral diagnostics using CRISPR‐Cas13. Science 360: 444–448.
24 Rees, H.A. and Liu, D.R. (2018). Base editing: precision chemistry on the genome and transcriptome of living cells. Nat. Rev. Genet. 19: 770–788.
25 Vamathevan, J., Clark, D., Czodrowski, P. et al. (2019). Applications of machine learning in drug discovery and development. Nat. Rev. Drug Discov. 18: 463–477.
26 Wiedenheft, B., Sternberg, S.H., and Doudna, J.A. (2012). RNA‐guided genetic silencing systems in bacteria and archaea. Nature 482: 331–338.
2 Historical Overview of Genome Editing from Bacteria to Higher Eukaryotes
Marcello Maresca
Genome Engineering, Discovery Sciences, BioPharmaceuticals R&D, AstraZeneca, Gothenburg, Sweden
2.1 Introduction
Molecular cloning methods have been instrumental for the establishment of the biotechnological industry. The ability to clone any DNA sequence of interest into a DNA vector has been a key technology advancement toward the generation of cellular and animal model of disease and the development of biopharmaceuticals. Traditional molecular cloning methods mostly rely on restriction enzymes‐mediated digestion СКАЧАТЬ