Literature DB >> 33609253

Mini review: genome and transcriptome editing using CRISPR-cas systems for haematological malignancy gene therapy.

Esther K Elliott1, Larisa M Haupt1, Lyn R Griffiths2.   

Abstract

The recent introduction of clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR associated protein (Cas) systems, offer an array of genome and transcriptome editing tools for clinical repair strategies. These include Cas9, Cas12a, dCas9 and more recently Cas13 effectors. RNA targeting CRISPR-Cas13 complexes show unique characteristics with the capability to engineer transcriptomes and modify gene expression, providing a potential clinical cancer therapy tool across various tissue types. Cas13 effectors such as RNA base editing for A to I replacement allows for precise transcript modification. Further applications of Cas13a highlights its capability of producing rapid diagnostic results in a mobile platform. This review will focus on the adaptions of existing CRISPR-Cas systems, along with new Cas effectors for transcriptome or RNA modifications used in disease modelling and gene therapy for haematological malignancy. We also address the current diagnostic and therapeutic potential of CRISPR-Cas systems for personalised haematological malignancy.

Entities:  

Keywords:  CRISPR1; Cas4; Gene-therapy3; Genome-editing2; Malignancy6; Transcriptome5

Mesh:

Year:  2021        PMID: 33609253     DOI: 10.1007/s11248-020-00232-9

Source DB:  PubMed          Journal:  Transgenic Res        ISSN: 0962-8819            Impact factor:   2.788


  51 in total

Review 1.  CRISPR-Cas systems in bacteria and archaea: versatile small RNAs for adaptive defense and regulation.

Authors:  Devaki Bhaya; Michelle Davison; Rodolphe Barrangou
Journal:  Annu Rev Genet       Date:  2011       Impact factor: 16.830

2.  Identification of preexisting adaptive immunity to Cas9 proteins in humans.

Authors:  Carsten T Charlesworth; Priyanka S Deshpande; Daniel P Dever; Joab Camarena; Viktor T Lemgart; M Kyle Cromer; Christopher A Vakulskas; Michael A Collingwood; Liyang Zhang; Nicole M Bode; Mark A Behlke; Beruh Dejene; Brandon Cieniewicz; Rosa Romano; Benjamin J Lesch; Natalia Gomez-Ospina; Sruthi Mantri; Mara Pavel-Dinu; Kenneth I Weinberg; Matthew H Porteus
Journal:  Nat Med       Date:  2019-01-28       Impact factor: 53.440

3.  Cardiologists send up a trial balloon in new efforts to relieve heart failure.

Authors:  M F Goldsmith
Journal:  JAMA       Date:  1987-01-16       Impact factor: 56.272

4.  [The efficacy and safety of second allogeneic hematopoietic stem cell transplantation for post-transplant hematologic malignancies relapse].

Authors:  Yu-Hong Chen; Lan-Ping Xu; Huan Chen; Dai-Hong Liu; Xiao-Hui Zhang; Wei Han; Feng-Rong Wang; Jing-Zhi Wang; Yu Wang; Ting Zhao; Yao Chen; Yuan-Yuan Zhang; Chen-Hua Yan; Yu-Qian Sun; Kai-Yan Liu; Xiao-Jun Huang
Journal:  Zhonghua Nei Ke Za Zhi       Date:  2011-06

5.  RNA targeting with CRISPR-Cas13.

Authors:  Omar O Abudayyeh; Jonathan S Gootenberg; Patrick Essletzbichler; Shuo Han; Julia Joung; Joseph J Belanto; Vanessa Verdine; David B T Cox; Max J Kellner; Aviv Regev; Eric S Lander; Daniel F Voytas; Alice Y Ting; Feng Zhang
Journal:  Nature       Date:  2017-10-04       Impact factor: 49.962

6.  Priming Human Repopulating Hematopoietic Stem and Progenitor Cells for Cas9/sgRNA Gene Targeting.

Authors:  Carsten T Charlesworth; Joab Camarena; M Kyle Cromer; Sriram Vaidyanathan; Rasmus O Bak; Jason M Carte; Jason Potter; Daniel P Dever; Matthew H Porteus
Journal:  Mol Ther Nucleic Acids       Date:  2018-05-03       Impact factor: 8.886

7.  Multiplexed activation of endogenous genes by CRISPR-on, an RNA-guided transcriptional activator system.

Authors:  Albert W Cheng; Haoyi Wang; Hui Yang; Linyu Shi; Yarden Katz; Thorold W Theunissen; Sudharshan Rangarajan; Chikdu S Shivalila; Daniel B Dadon; Rudolf Jaenisch
Journal:  Cell Res       Date:  2013-08-27       Impact factor: 25.617

8.  Exome Sequencing Diagnoses X-Linked Moesin-Associated Immunodeficiency in a Primary Immunodeficiency Case.

Authors:  Gabrielle Bradshaw; Robbie R Lualhati; Cassie L Albury; Neven Maksemous; Deidre Roos-Araujo; Robert A Smith; Miles C Benton; David A Eccles; Rod A Lea; Heidi G Sutherland; Larisa M Haupt; Lyn R Griffiths
Journal:  Front Immunol       Date:  2018-03-05       Impact factor: 7.561

9.  An "off-the-shelf" fratricide-resistant CAR-T for the treatment of T cell hematologic malignancies.

Authors:  Matthew L Cooper; Jaebok Choi; Karl Staser; Julie K Ritchey; Jessica M Devenport; Kayla Eckardt; Michael P Rettig; Bing Wang; Linda G Eissenberg; Armin Ghobadi; Leah N Gehrs; Julie L Prior; Samuel Achilefu; Christopher A Miller; Catrina C Fronick; Julie O'Neal; Feng Gao; David M Weinstock; Alejandro Gutierrez; Robert S Fulton; John F DiPersio
Journal:  Leukemia       Date:  2018-02-20       Impact factor: 11.528

10.  Search-and-replace genome editing without double-strand breaks or donor DNA.

Authors:  Andrew V Anzalone; Peyton B Randolph; Jessie R Davis; Alexander A Sousa; Luke W Koblan; Jonathan M Levy; Peter J Chen; Christopher Wilson; Gregory A Newby; Aditya Raguram; David R Liu
Journal:  Nature       Date:  2019-10-21       Impact factor: 69.504

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  2 in total

Review 1.  CRISPR-Cas systems target endogenous genes to impact bacterial physiology and alter mammalian immune responses.

Authors:  Qun Wu; Luqing Cui; Yingying Liu; Rongpeng Li; Menghong Dai; Zhenwei Xia; Min Wu
Journal:  Mol Biomed       Date:  2022-07-20

Review 2.  Utilization of CRISPR-Mediated Tools for Studying Functional Genomics in Hematological Malignancies: An Overview on the Current Perspectives, Challenges, and Clinical Implications.

Authors:  Maheswaran Solayappan; Adam Azlan; Kang Zi Khor; Mot Yee Yik; Matiullah Khan; Narazah Mohd Yusoff; Emmanuel Jairaj Moses
Journal:  Front Genet       Date:  2022-01-28       Impact factor: 4.599

  2 in total

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