A guide for drug inducible transcriptional activation with HIT systems.

Precise investigation and manipulation of gene function often require modulation in a controlled and dynamic manner. In this chapter, we describe the methods to apply HIT systems for drug inducible transcriptional activation or simultaneous activation and genome editing in human cells. Together with those for editing, which are described in another chapter, HIT systems herein provide a valuable toolbox toward many biological applications, especially when precision and dynamics are required for a functional perturbation.

[1]  Christopher M. Vockley,et al.  RNA-guided gene activation by CRISPR-Cas9-based transcription factors , 2013, Nature Methods.

[2]  Morgan L. Maeder,et al.  CRISPR RNA-guided activation of endogenous human genes , 2013, Nature Methods.

[3]  N. Perrimon,et al.  Comparative Analysis of Cas9 Activators Across Multiple Species , 2016, Nature Methods.

[4]  Ronald D. Vale,et al.  A Protein-Tagging System for Signal Amplification in Gene Expression and Fluorescence Imaging , 2014, Cell.

[5]  P Chambon,et al.  Regulation of Cre recombinase activity by mutated estrogen receptor ligand-binding domains. , 1997, Biochemical and biophysical research communications.

[6]  Jia Lu,et al.  HIT-Cas9: A CRISPR/Cas9 Genome-Editing Device under Tight and Effective Drug Control , 2018, Molecular therapy. Nucleic acids.

[7]  C. Branda,et al.  Talking about a revolution: The impact of site-specific recombinases on genetic analyses in mice. , 2004, Developmental cell.

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

[9]  Chad A. Cowan,et al.  Enhanced efficiency of human pluripotent stem cell genome editing through replacing TALENs with CRISPRs. , 2013, Cell stem cell.

[10]  P. Chambon,et al.  Conditional site-specific recombination in mammalian cells using a ligand-dependent chimeric Cre recombinase. , 1995, Proceedings of the National Academy of Sciences of the United States of America.

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

[12]  Donghai Wu,et al.  Comparison of TALE designer transcription factors and the CRISPR/dCas9 in regulation of gene expression by targeting enhancers , 2014, Nucleic acids research.

[13]  Matthew J. Moscou,et al.  A Simple Cipher Governs DNA Recognition by TAL Effectors , 2009, Science.

[14]  N. Perrimon,et al.  Highly-efficient Cas9-mediated transcriptional programming , 2015, Nature Methods.

[15]  H. Leonhardt,et al.  Targeted transcriptional activation of silent oct4 pluripotency gene by combining designer TALEs and inhibition of epigenetic modifiers , 2012, Nucleic acids research.

[16]  J. Keith Joung,et al.  TALENs: a widely applicable technology for targeted genome editing , 2012, Nature Reviews Molecular Cell Biology.

[17]  Jens Boch,et al.  Breaking the Code of DNA Binding Specificity of TAL-Type III Effectors , 2009, Science.

[18]  J. Vogel,et al.  CRISPR RNA maturation by trans-encoded small RNA and host factor RNase III , 2011, Nature.

[19]  G. Church,et al.  CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering , 2013, Nature Biotechnology.

[20]  G. Church,et al.  Efficient construction of sequence-specific TAL effectors for modulating mammalian transcription. , 2011, Nature biotechnology.

[21]  An Xiao,et al.  Heritable gene targeting in zebrafish using customized TALENs , 2011, Nature Biotechnology.

[22]  Yarden Katz,et al.  Multiplexed activation of endogenous genes by CRISPR-on, an RNA-guided transcriptional activator system , 2013, Cell Research.

[23]  Feng Zhang,et al.  Orthogonal gene knock out and activation with a catalytically active Cas9 nuclease , 2015, Nature Biotechnology.

[24]  Jia Lu,et al.  Multimode drug inducible CRISPR/Cas9 devices for transcriptional activation and genome editing , 2017, Nucleic acids research.

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

[26]  Luke A. Gilbert,et al.  CRISPR-Mediated Modular RNA-Guided Regulation of Transcription in Eukaryotes , 2013, Cell.

[27]  G. Church,et al.  Cas9 gRNA engineering for genome editing, activation and repression , 2015, Nature Methods.

[28]  E. Lander,et al.  Development and Applications of CRISPR-Cas9 for Genome Engineering , 2014, Cell.

[29]  Elo Leung,et al.  A TALE nuclease architecture for efficient genome editing , 2011, Nature Biotechnology.

[30]  G. Church,et al.  Cas9 as a versatile tool for engineering biology , 2013, Nature Methods.

[31]  P Chambon,et al.  Temporally-controlled site-specific mutagenesis in the basal layer of the epidermis: comparison of the recombinase activity of the tamoxifen-inducible Cre-ER(T) and Cre-ER(T2) recombinases. , 1999, Nucleic acids research.

[32]  Y. Ying,et al.  Multiple Chemical Inducible Tal Effectors for Genome Editing and Transcription Activation. , 2018, ACS chemical biology.