Literature DB >> 30803822

Genome Editing with mRNA Encoding ZFN, TALEN, and Cas9.

Hong-Xia Zhang1, Ying Zhang2, Hao Yin3.   

Abstract

Genome-editing technologies based on programmable nucleases have significantly broadened our ability to make precise and direct changes in the genomic DNA of various species, including human cells. Delivery of programmable nucleases into the target tissue or cell is one of the pressing challenges in transforming the technology into medicine. In vitro-transcribed (IVT) mRNA-mediated delivery of nucleases has several advantages, such as transient expression with efficient in vivo and in vitro delivery, no genomic integration, a potentially low off-target rate, and high editing efficiency. This review focuses on key barriers related to IVT mRNA delivery, on developed modes of delivery, and on the application and future prospects of mRNA encoding nuclease-mediated genome editing in research and clinical trials.
Copyright © 2019 The American Society of Gene and Cell Therapy. Published by Elsevier Inc. All rights reserved.

Entities:  

Year:  2019        PMID： 30803822      PMCID： PMC6453514          DOI： 10.1016/j.ymthe.2019.01.014

Source DB:  PubMed          Journal:  Mol Ther        ISSN： 1525-0016            Impact factor:   11.454


  162 in total

Review 1.  Quantum dots for live cells, in vivo imaging, and diagnostics.

Authors:  X Michalet; F F Pinaud; L A Bentolila; J M Tsay; S Doose; J J Li; G Sundaresan; A M Wu; S S Gambhir; S Weiss
Journal:  Science       Date:  2005-01-28       Impact factor: 47.728

2.  In vivo genome editing using nuclease-encoding mRNA corrects SP-B deficiency.

Authors:  Azita J Mahiny; Alexander Dewerth; Lauren E Mays; Mohammed Alkhaled; Benedikt Mothes; Emad Malaeksefat; Brigitta Loretz; Jennifer Rottenberger; Darina M Brosch; Philipp Reautschnig; Pacharapan Surapolchai; Franziska Zeyer; Andrea Schams; Melanie Carevic; Martina Bakele; Matthias Griese; Matthias Schwab; Bernd Nürnberg; Sandra Beer-Hammer; Rupert Handgretinger; Dominik Hartl; Claus-Michael Lehr; Michael S D Kormann
Journal:  Nat Biotechnol       Date:  2015-05-18       Impact factor: 54.908

Review 3.  Cationic lipids, lipoplexes and intracellular delivery of genes.

Authors:  Luc Wasungu; Dick Hoekstra
Journal:  J Control Release       Date:  2006-06-28       Impact factor: 9.776

4.  Efficient modification of CCR5 in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template.

Authors:  Blythe D Sather; Guillermo S Romano Ibarra; Karen Sommer; Gabrielle Curinga; Malika Hale; Iram F Khan; Swati Singh; Yumei Song; Kamila Gwiazda; Jaya Sahni; Jordan Jarjour; Alexander Astrakhan; Thor A Wagner; Andrew M Scharenberg; David J Rawlings
Journal:  Sci Transl Med       Date:  2015-09-30       Impact factor: 17.956

5.  Correction of a genetic disease by CRISPR-Cas9-mediated gene editing in mouse spermatogonial stem cells.

Authors:  Yuxuan Wu; Hai Zhou; Xiaoying Fan; Ying Zhang; Man Zhang; Yinghua Wang; Zhenfei Xie; Meizhu Bai; Qi Yin; Dan Liang; Wei Tang; Jiaoyang Liao; Chikai Zhou; Wujuan Liu; Ping Zhu; Hongshan Guo; Hong Pan; Chunlian Wu; Huijuan Shi; Ligang Wu; Fuchou Tang; Jinsong Li
Journal:  Cell Res       Date:  2014-12-05       Impact factor: 25.617

6.  Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV.

Authors:  Pablo Tebas; David Stein; Winson W Tang; Ian Frank; Shelley Q Wang; Gary Lee; S Kaye Spratt; Richard T Surosky; Martin A Giedlin; Geoff Nichol; Michael C Holmes; Philip D Gregory; Dale G Ando; Michael Kalos; Ronald G Collman; Gwendolyn Binder-Scholl; Gabriela Plesa; Wei-Ting Hwang; Bruce L Levine; Carl H June
Journal:  N Engl J Med       Date:  2014-03-06       Impact factor: 91.245

7.  Increased erythropoiesis in mice injected with submicrogram quantities of pseudouridine-containing mRNA encoding erythropoietin.

Authors:  Katalin Karikó; Hiromi Muramatsu; Jason M Keller; Drew Weissman
Journal:  Mol Ther       Date:  2012-02-14       Impact factor: 11.454

Review 8.  Meganucleases and other tools for targeted genome engineering: perspectives and challenges for gene therapy.

Authors:  George Silva; Laurent Poirot; Roman Galetto; Julianne Smith; Guillermo Montoya; Philippe Duchateau; Frédéric Pâques
Journal:  Curr Gene Ther       Date:  2011-02       Impact factor: 4.391

9.  Efficient genome editing in zebrafish using a CRISPR-Cas system.

Authors:  Woong Y Hwang; Yanfang Fu; Deepak Reyon; Morgan L Maeder; Shengdar Q Tsai; Jeffry D Sander; Randall T Peterson; J-R Joanna Yeh; J Keith Joung
Journal:  Nat Biotechnol       Date:  2013-01-29       Impact factor: 54.908

10.  Highly efficient homology-driven genome editing in human T cells by combining zinc-finger nuclease mRNA and AAV6 donor delivery.

Authors:  Jianbin Wang; Joshua J DeClercq; Samuel B Hayward; Patrick Wai-Lun Li; David A Shivak; Philip D Gregory; Gary Lee; Michael C Holmes
Journal:  Nucleic Acids Res       Date:  2015-11-02       Impact factor: 16.971
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  33 in total

1.  In vitro-Transcribed mRNA Therapeutics: Out of the Shadows and Into the Spotlight.

Authors:  Katalin Karikó
Journal:  Mol Ther       Date:  2019-03-21       Impact factor: 11.454

Review 2.  RNA delivery biomaterials for the treatment of genetic and rare diseases.

Authors:  Weiyu Zhao; Xucheng Hou; Olivia G Vick; Yizhou Dong
Journal:  Biomaterials       Date:  2019-06-20       Impact factor: 12.479

Review 3.  Gene-Modified Stem Cells for Spinal Cord Injury: a Promising Better Alternative Therapy.

Authors:  Yirui Feng; Yu Li; Ping-Ping Shen; Bin Wang
Journal:  Stem Cell Rev Rep       Date:  2022-05-19       Impact factor: 5.739

Review 4.  Genome centric engineering using ZFNs, TALENs and CRISPR-Cas9 systems for trait improvement and disease control in Animals.

Authors:  Atif Khurshid Wani; Nahid Akhtar; Reena Singh; Ajit Prakash; Sayed Haidar Abbas Raza; Simona Cavalu; Chirag Chopra; Mahmoud Madkour; Ahmed Elolimy; Nesrein M Hashem
Journal:  Vet Res Commun       Date:  2022-07-04       Impact factor: 2.459

Review 5.  mRNA-based therapeutics: powerful and versatile tools to combat diseases.

Authors:  Shugang Qin; Xiaoshan Tang; Yuting Chen; Kepan Chen; Na Fan; Wen Xiao; Qian Zheng; Guohong Li; Yuqing Teng; Min Wu; Xiangrong Song
Journal:  Signal Transduct Target Ther       Date:  2022-05-21

6.  Nanodelivery of nucleic acids.

Authors:  Bárbara B Mendes; João Conniot; Aviram Avital; Dongbao Yao; Xingya Jiang; Xiang Zhou; Noga Sharf-Pauker; Yuling Xiao; Omer Adir; Haojun Liang; Jinjun Shi; Avi Schroeder; João Conde
Journal:  Nat Rev Methods Primers       Date:  2022-04-14

Review 7.  Gene transfer to plants by electroporation: methods and applications.

Authors:  Ibrahim Ilker Ozyigit
Journal:  Mol Biol Rep       Date:  2020-04-02       Impact factor: 2.316

Review 8.  Genome-editing approaches and applications: a brief review on CRISPR technology and its role in cancer.

Authors:  Narmadhaa Siva; Sonal Gupta; Ayam Gupta; Jayendra Nath Shukla; Babita Malik; Nidhi Shukla
Journal:  3 Biotech       Date:  2021-02-26       Impact factor: 2.406

Review 9.  Understanding and overcoming adverse consequences of genome editing on hematopoietic stem and progenitor cells.

Authors:  Byung-Chul Lee; Richard J Lozano; Cynthia E Dunbar
Journal:  Mol Ther       Date:  2021-09-10       Impact factor: 11.454

Review 10.  A Revolution toward Gene-Editing Technology and Its Application to Crop Improvement.

Authors:  Sunny Ahmar; Sumbul Saeed; Muhammad Hafeez Ullah Khan; Shahid Ullah Khan; Freddy Mora-Poblete; Muhammad Kamran; Aroosha Faheem; Ambreen Maqsood; Muhammad Rauf; Saba Saleem; Woo-Jong Hong; Ki-Hong Jung
Journal:  Int J Mol Sci       Date:  2020-08-07       Impact factor: 5.923
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