Nuclease-Deficient Clustered Regularly Interspaced Short Palindromic Repeat-Based Approaches for In Vitro and In Vivo Gene Activation

被引：2

作者：

Lek, Angela ^{[1
]}

Ma, Kaiyue ^{[1
]}

Woodman, Keryn G. ^{[1
]}

Lek, Monkol ^{[1
]}

机构：

[1] Yale Sch Med, Dept Genet, 300 Cedar St,TACS341C, New Haven, CT 06520 USA

来源：

HUMAN GENE THERAPY | 2021年 / 32卷 / 5-6期

关键词：

CRISPR; gene activation; gene upregulation;

D O I：

10.1089/hum.2020.241

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

Clustered regularly interspaced short palindromic repeat (CRISPR)-based technology has been adapted to achieve a wide range of genome modifications, including transcription regulation. The focus of this review is on the application of CRISPR-based platforms such as nuclease-deficient Cas9 and Cas12a, to achieve targeted gene activation. We review studies to date that have used CRISPR-based activation technology for the elucidation of biological mechanism and disease correction, as well as its application in genetic screens as a powerful tool for high-throughput genotype-phenotype mapping. In addition to our synthesis and critical analysis of published studies, we explore key considerations for the potential clinical translation of CRISPR-based activation technology.

引用

页码：260 / 274

页数：15

共 95 条

[1] A Single-Cell Transcriptomics CRISPR-Activation Screen Identifies Epigenetic Regulators of the Zygotic Genome Activation Program [J].

Alda-Catalinas, Celia ;

Bredikhin, Danila ;

Hernando-Herraez, Irene ;

Santos, Fatima ;

Kubinyecz, Oana ;

Eckersley-Maslin, Melanie A. ;

Stegle, Oliver ;

Reik, Wolf .

CELL SYSTEMS, 2020, 11 (01) :25-+

[2] High-dose AAV gene therapy deaths [J].

不详 .

NATURE BIOTECHNOLOGY, 2020, 38 (08) :910-910

[3] Conditionally Stabilized dCas9 Activator for Controlling Gene Expression in Human Cell Reprogramming and Differentiation [J].

Balboa, Diego ;

Weltner, Jere ;

Eurola, Solja ;

Trokovic, Ras ;

Wartiovaara, Kirmo ;

Otonkoski, Timo .

STEM CELL REPORTS, 2015, 5 (03) :448-459

[4] CRISPR provides acquired resistance against viruses in prokaryotes [J].

Barrangou, Rodolphe ;

Fremaux, Christophe ;

Deveau, Helene ;

Richards, Melissa ;

Boyaval, Patrick ;

Moineau, Sylvain ;

Romero, Dennis A. ;

Horvath, Philippe .

SCIENCE, 2007, 315 (5819) :1709-1712

[5] Targeted Epigenetic Remodeling of Endogenous Loci by CRISPR/Cas9-Based Transcriptional Activators Directly Converts Fibroblasts to Neuronal Cells [J].

Black, Joshua B. ;

Adler, Andrew F. ;

Wang, Hong-Gang ;

D'Ippolito, Anthony M. ;

Hutchinson, Hunter A. ;

Reddy, Timothy E. ;

Pitt, Geoffrey S. ;

Leong, Kam W. ;

Gersbach, Charles A. .

CELL STEM CELL, 2016, 19 (03) :406-414

[6] Writing of H3K4Me3 overcomes epigenetic silencing in a sustained but context-dependent manner [J].

Cano-Rodriguez, David ;

Gjaltema, Rutger A. F. ;

Jilderda, Laura J. ;

Jellema, Pytrick ;

Dokter-Fokkens, Jelleke ;

Ruiters, Marcel H. J. ;

Rots, Marianne G. .

NATURE COMMUNICATIONS, 2016, 7

[7] CRISPR-Cas orthologues and variants: optimizing the repertoire, specificity and delivery of genome engineering tools [J].

Cebrian-Serrano, Alberto ;

Davies, Benjamin .

MAMMALIAN GENOME, 2017, 28 (7-8) :247-261

[8] A CRISPR/Cas9-Based System for Reprogramming Cell Lineage Specification [J].

Chakraborty, Syandan ;

Ji, HaYeun ;

Kabadi, Ami M. ;

Gersbach, Charles A. ;

Christoforou, Nicolas ;

Leong, Kam W. .

STEM CELL REPORTS, 2014, 3 (06) :940-947

[9]

Chavez A, 2016, NAT METHODS, V13, P563, DOI [10.1038/NMETH.3871, 10.1038/nmeth.3871]

[10] Randomized CRISPR-Cas Transcriptional Perturbation Screening Reveals Protective Genes against Alpha-Synuclein Toxicity [J].

Chen, Ying-Chou ;

Farzadfard, Fahim ;

Gharaei, Nava ;

Chen, William C. W. ;

Cao, Jicong ;

Lu, Timothy K. .

MOLECULAR CELL, 2017, 68 (01) :247-+

← 1 2 3 4 5 6 7 8 9 10 →