CRISPR/Cas9 – An evolving biological tool kit for cancer biology and oncology

The development of genetic engineering in the 1970s marked a new frontier in genome-editing technology. Gene-editing technologies have provided a plethora of benefits to the life sciences. The clustered regularly interspaced short palindromic repeats/CRISPR associated protein 9 (CRISPR/ Cas9) system is a versatile technology that provides the ability to add or remove DNA in the genome in a sequence-specific manner. Serious efforts are underway to improve the efficiency of CRISPR/Cas9 targeting and thus reduce off-target effects. Currently, various applications of CRISPR/Cas9 are used in cancer biology and oncology to perform robust site-specific gene editing, thereby becoming more useful for biological and clinical applications. Many variants and applications of CRISPR/Cas9 are being rapidly developed. Experimental approaches that are based on CRISPR technology have created a very promising tool that is inexpensive and simple for developing effective cancer therapeutics. This review discusses diverse applications of CRISPR-based gene-editing tools in oncology and potential future cancer therapies.

[1]  Howard Y. Chang,et al.  NONCODING RNA: CRISPRi‐based genome‐scale identification of functional long noncoding RNA loci in human cells , 2017 .

[2]  Gabriella Ficz,et al.  Hit-and-run epigenetic editing prevents senescence entry in primary breast cells from healthy donors , 2017, Nature Communications.

[3]  C. Barbas,et al.  ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering. , 2013, Trends in biotechnology.

[4]  Sojib Bin Zaman,et al.  CRISPR-Cas9: a promising genetic engineering approach in cancer research , 2018, Therapeutic advances in medical oncology.

[5]  Ioana Berindan-Neagoe,et al.  CRISPR/Cas9: A Potential Life-Saving Tool. What’s next? , 2017, Molecular therapy. Nucleic acids.

[6]  J. Shrager,et al.  CRISPR/Cas‐mediated genome editing to treat EGFR‐mutant lung cancer: a personalized molecular surgical therapy , 2016, EMBO molecular medicine.

[7]  Aviv Regev,et al.  Generation of mouse models of myeloid malignancy with combinatorial genetic lesions using CRISPR-Cas9 genome editing , 2014, Nature Biotechnology.

[8]  David Cyranoski,et al.  CRISPR gene-editing tested in a person for the first time , 2016, Nature.

[9]  Li Zhang,et al.  MiR-152 suppresses the proliferation and invasion of NSCLC cells by inhibiting FGF2 , 2014, Experimental & Molecular Medicine.

[10]  Ding Ma,et al.  Disruption of HPV16-E7 by CRISPR/Cas System Induces Apoptosis and Growth Inhibition in HPV16 Positive Human Cervical Cancer Cells , 2014, BioMed research international.

[11]  Nikhil U. Nair,et al.  Toward a genetic tool development pipeline for host-associated bacteria. , 2017, Current opinion in microbiology.

[12]  Feng Zhang,et al.  Crystal Structure of Cas9 in Complex with Guide RNA and Target DNA , 2014, Cell.

[13]  E. Rebar,et al.  Genome editing with engineered zinc finger nucleases , 2010, Nature Reviews Genetics.

[14]  W. Cho,et al.  肺癌的个体化靶向治疗 , 2013, Zhongguo fei ai za zhi = Chinese journal of lung cancer.

[15]  Alexandre Hiroaki Kihara,et al.  CRISPR, Prime Editing, Optogenetics, and DREADDs: New Therapeutic Approaches Provided by Emerging Technologies in the Treatment of Spinal Cord Injury , 2020, Molecular Neurobiology.

[16]  Aviv Regev,et al.  RNA targeting with CRISPR–Cas13 , 2017, Nature.

[17]  P. Renz,et al.  CRISPR-mediated genotypic and phenotypic correction of a chronic granulomatous disease mutation in human iPS cells , 2015, Experimental hematology.

[18]  Neville E. Sanjana,et al.  Improved vectors and genome-wide libraries for CRISPR screening , 2014, Nature Methods.

[19]  Michael T. McManus,et al.  Choosing the Right Tool for the Job: RNAi, TALEN, or CRISPR. , 2015, Molecular cell.

[20]  Timothy K Lu,et al.  Multiplexed barcoded CRISPR-Cas9 screening enabled by CombiGEM , 2016, Proceedings of the National Academy of Sciences.

[21]  Max A. Horlbeck,et al.  Genome-Scale CRISPR-Mediated Control of Gene Repression and Activation , 2014, Cell.

[22]  William Sun,et al.  CRISPR-mediated targeting of HER2 inhibits cell proliferation through a dominant negative mutation. , 2017, Cancer letters.

[23]  Hao Yin,et al.  Precision cancer mouse models through genome editing with CRISPR-Cas9 , 2015, Genome Medicine.

[24]  Hidde L Ploegh,et al.  Inhibition of non-homologous end joining increases the efficiency of CRISPR/Cas9-mediated precise [TM: inserted] genome editing , 2015, Nature Biotechnology.

[25]  A. Laurenzana,et al.  Type II CRISPR/Cas9 approach in the oncological therapy , 2017, Journal of Experimental & Clinical Cancer Research.

[26]  J. Doudna,et al.  A Programmable Dual-RNA–Guided DNA Endonuclease in Adaptive Bacterial Immunity , 2012, Science.

[27]  Michael P Snyder,et al.  Personalized sequencing and the future of medicine: discovery, diagnosis and defeat of disease. , 2014, Pharmacogenomics.

[28]  Steven Lin,et al.  Enhanced homology-directed human genome engineering by controlled timing of CRISPR/Cas9 delivery , 2014, eLife.

[29]  Y. Wang,et al.  The BRCA1-Δ11q Alternative Splice Isoform Bypasses Germline Mutations and Promotes Therapeutic Resistance to PARP Inhibition and Cisplatin. , 2016, Cancer research.

[30]  Gaelen T. Hess,et al.  Synergistic drug combinations for cancer identified in a CRISPR screen for pairwise genetic interactions , 2017, Nature Biotechnology.

[31]  A. McKenna,et al.  Genetic and Clonal Dissection of Murine Small Cell Lung Carcinoma Progression by Genome Sequencing , 2014, Cell.

[32]  John P. Overington,et al.  The promise and peril of chemical probes. , 2015, Nature chemical biology.

[33]  Kamel Khalili,et al.  CRISPR/Cas9 and cancer targets: future possibilities and present challenges , 2016, Oncotarget.

[34]  A. Ashworth,et al.  Genetic Interactions in Cancer Progression and Treatment , 2011, Cell.

[35]  Rohit Bhargava,et al.  MAN2A1-FER Fusion Gene Is Expressed by Human Liver and Other Tumor Types and Has Oncogenic Activity in Mice. , 2017, Gastroenterology.

[36]  Henning Budde,et al.  CRISPR/Cas9, the Powerful New Genome-Editing Tool for Putative Therapeutics in Obesity , 2018, Journal of Molecular Neuroscience.

[37]  Silvia Liu,et al.  Targeting genomic rearrangements in tumor cells using Cas9-mediated insertion of a suicide gene , 2017, Nature Biotechnology.

[38]  E. Baloglu,et al.  Target identification of small molecules using large-scale CRISPR-Cas mutagenesis scanning of essential genes , 2018, Nature Communications.

[39]  George P. Lisi,et al.  1H, 13C, 15N backbone and side chain resonance assignment of the HNH nuclease from Streptococcus pyogenes CRISPR-Cas9 , 2019, Biomolecular NMR Assignments.

[40]  Kosei Maemura,et al.  Molecular mechanism of cholangiocarcinoma carcinogenesis , 2014, Journal of hepato-biliary-pancreatic sciences.

[41]  Jennifer A. Doudna,et al.  CRISPR-Cas12a target binding unleashes indiscriminate single-stranded DNase activity , 2018, Science.

[42]  K. Wimmer,et al.  Neurofibromatosis type 1 (NF1) and Associated Tumors , 2014, Klinische Pädiatrie.

[43]  Xueling Wu,et al.  Cas9 Protein Triggers Differential Expression of Inherent Genes Especially NGFR Expression in 293T Cells , 2019, Cellular and Molecular Bioengineering.

[44]  Robert Kammerer,et al.  Potent induction of B- and T-cell immunity against human carcinoembryonic antigen-expressing tumors in human carcinoembryonic antigen transgenic mice mediated by direct lentivector injection , 2009, Molecular Cancer Therapeutics.

[45]  Byung-Joo Ham,et al.  Specificity Assessment of CRISPR Genome Editing of Oncogenic EGFR Point Mutation with Single-Base Differences , 2019, Molecules.

[46]  Neville E. Sanjana,et al.  High-throughput functional genomics using CRISPR–Cas9 , 2015, Nature Reviews Genetics.

[47]  J. Keith Joung,et al.  High frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells , 2013, Nature Biotechnology.

[48]  Eugene V Koonin,et al.  CRISPR-Cas , 2013, RNA biology.

[49]  G. Novelli,et al.  Application of CRISPR/Cas9 to human-induced pluripotent stem cells: from gene editing to drug discovery , 2020, Human Genomics.

[50]  Ying Ding,et al.  Novel fusion transcripts associate with progressive prostate cancer. , 2014, The American journal of pathology.

[51]  James E. DiCarlo,et al.  RNA-Guided Human Genome Engineering via Cas9 , 2013, Science.

[52]  David A. Scott,et al.  Double Nicking by RNA-Guided CRISPR Cas9 for Enhanced Genome Editing Specificity , 2013, Cell.

[53]  Daniel S. Chertow,et al.  Next-generation diagnostics with CRISPR , 2018, Science.

[54]  Bin Zhang,et al.  Applications of TALENs and CRISPR/Cas9 in Human Cells and Their Potentials for Gene Therapy , 2014, Molecular Biotechnology.

[55]  Hakho Lee,et al.  Genome-wide CRISPR Screen in a Mouse Model of Tumor Growth and Metastasis , 2015, Cell.

[56]  Aviv Regev,et al.  Nucleic acid detection with CRISPR-Cas13a/C2c2 , 2017, Science.

[57]  Y. Shentu,et al.  Pembrolizumab versus Chemotherapy for PD-L1-Positive Non-Small-Cell Lung Cancer. , 2016, The New England journal of medicine.

[58]  R. Bernards,et al.  CRISPR knockout screening outperforms shRNA and CRISPRi in identifying essential genes , 2016, Nature Biotechnology.

[59]  N. Rahman,et al.  Mainstreaming genetic testing of cancer predisposition genes. , 2014, Clinical medicine.

[60]  Olivier Elemento,et al.  DrugTargetSeqR: a genomics- and CRISPR/Cas9-based method to analyze drug targets , 2014, Nature chemical biology.

[61]  Luke A. Gilbert,et al.  CRISPR interference (CRISPRi) for sequence-specific control of gene expression , 2013, Nature Protocols.

[62]  T. Papagiannakopoulos,et al.  Application of CRISPR-mediated genome engineering in cancer research. , 2017, Cancer letters.

[63]  Tian Xu,et al.  The expanding role of mouse genetics for understanding human biology and disease , 2008, Disease Models & Mechanisms.

[64]  Takanori Kanai,et al.  Modeling colorectal cancer using CRISPR-Cas9–mediated engineering of human intestinal organoids , 2015, Nature Medicine.

[65]  Demetrios A. Spandidos,et al.  CRISPR therapeutic tools for complex genetic disorders and cancer (Review) , 2018, International journal of oncology.

[66]  J. Rinn,et al.  An Integrated Genome-wide CRISPRa Approach to Functionalize lncRNAs in Drug Resistance , 2018, Cell.

[67]  J. Kinney,et al.  Discovery of cancer drug targets by CRISPR-Cas9 screening of protein domains , 2015, Nature Biotechnology.

[68]  B Gryseels,et al.  Drug resistance in human helminths: current situation and lessons from livestock. , 2000, Clinical microbiology reviews.

[69]  Francisco J. Sánchez-Rivera,et al.  Applications of the CRISPR–Cas9 system in cancer biology , 2015, Nature Reviews Cancer.

[70]  Ji-Lin Wang,et al.  MicroRNA 345, a methylation-sensitive microRNA is involved in cell proliferation and invasion in human colorectal cancer. , 2011, Carcinogenesis.

[71]  Kristen E. Pauken,et al.  The role of the PD‐1 pathway in autoimmunity and peripheral tolerance , 2011, Annals of the New York Academy of Sciences.

[72]  Le Cong,et al.  Multiplex Genome Engineering Using CRISPR/Cas Systems , 2013, Science.

[73]  Zhiping Weng,et al.  Genome-Wide CRISPR Screen Identifies Regulators of Mitogen-Activated Protein Kinase as Suppressors of Liver Tumors in Mice. , 2017, Gastroenterology.

[74]  Michael Boutros,et al.  CRISPR/Cas9 for cancer research and therapy. , 2019, Seminars in cancer biology.

[75]  Lenka Munoz,et al.  Non-kinase targets of protein kinase inhibitors , 2017, Nature Reviews Drug Discovery.

[76]  M. Kampmann.  CRISPRi and CRISPRa Screens in Mammalian Cells for Precision Biology and Medicine. , 2018, ACS chemical biology.

[77]  Haitao Yang,et al.  Break Breast Cancer Addiction by CRISPR/Cas9 Genome Editing , 2018, Journal of Cancer.