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Literature DB >> 28337020

Delivery technologies for genome editing.

Hao Yin¹, Kevin J Kauffman^1,2, Daniel G Anderson^1,2,3,4.

Abstract

With the recent development of CRISPR technology, it is becoming increasingly easy to engineer the genome. Genome-editing systems based on CRISPR, as well as transcription activator-like effector nucleases (TALENs) and zinc-finger nucleases (ZFNs), are becoming valuable tools for biomedical research, drug discovery and development, and even gene therapy. However, for each of these systems to effectively enter cells of interest and perform their function, efficient and safe delivery technologies are needed. This Review discusses the principles of biomacromolecule delivery and gene editing, examines recent advances and challenges in non-viral and viral delivery methods, and highlights the status of related clinical trials.

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Year: 2017 PMID： 28337020 DOI： 10.1038/nrd.2016.280

Source DB: PubMed Journal: Nat Rev Drug Discov ISSN： 1474-1776 Impact factor: 84.694

132 in total

1. Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity.

Authors: F Ann Ran; Patrick D Hsu; Chie-Yu Lin; Jonathan S Gootenberg; Silvana Konermann; Alexandro E Trevino; David A Scott; Azusa Inoue; Shogo Matoba; Yi Zhang; Feng Zhang
Journal: Cell Date: 2013-08-29 Impact factor: 41.582

2. RNAi-nanoparticulate manipulation of gene expression as a new functional genomics tool in the liver.

Authors: Hao Yin; Roman L Bogorad; Carmen Barnes; Stephen Walsh; Iris Zhuang; Hidenori Nonaka; Vera Ruda; Satya Kuchimanchi; Lubomir Nechev; Akin Akinc; Wen Xue; Marino Zerial; Robert Langer; Daniel G Anderson; Victor Koteliansky
Journal: J Hepatol Date: 2015-11-30 Impact factor: 25.083

3. Rational design of a split-Cas9 enzyme complex.

Authors: Addison V Wright; Samuel H Sternberg; David W Taylor; Brett T Staahl; Jorge A Bardales; Jack E Kornfeld; Jennifer A Doudna
Journal: Proc Natl Acad Sci U S A Date: 2015-02-23 Impact factor: 11.205

4. Efficient delivery of genome-editing proteins using bioreducible lipid nanoparticles.

Authors: Ming Wang; John A Zuris; Fantao Meng; Holly Rees; Shuo Sun; Pu Deng; Yong Han; Xue Gao; Dimitra Pouli; Qi Wu; Irene Georgakoudi; David R Liu; Qiaobing Xu
Journal: Proc Natl Acad Sci U S A Date: 2016-02-29 Impact factor: 11.205

5. In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy.

Authors: Christopher E Nelson; Chady H Hakim; David G Ousterout; Pratiksha I Thakore; Eirik A Moreb; Ruth M Castellanos Rivera; Sarina Madhavan; Xiufang Pan; F Ann Ran; Winston X Yan; Aravind Asokan; Feng Zhang; Dongsheng Duan; Charles A Gersbach
Journal: Science Date: 2015-12-31 Impact factor: 47.728

6. Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells.

Authors: Ayal Hendel; Rasmus O Bak; Joseph T Clark; Andrew B Kennedy; Daniel E Ryan; Subhadeep Roy; Israel Steinfeld; Benjamin D Lunstad; Robert J Kaiser; Alec B Wilkens; Rosa Bacchetta; Anya Tsalenko; Douglas Dellinger; Laurakay Bruhn; Matthew H Porteus
Journal: Nat Biotechnol Date: 2015-06-29 Impact factor: 54.908

7. 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

8. Autonomous zinc-finger nuclease pairs for targeted chromosomal deletion.

Authors: Cem Söllü; Kaweh Pars; Tatjana I Cornu; Stacey Thibodeau-Beganny; Morgan L Maeder; J Keith Joung; Regine Heilbronn; Toni Cathomen
Journal: Nucleic Acids Res Date: 2010-08-16 Impact factor: 16.971

9. CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes.

Authors: Puping Liang; Yanwen Xu; Xiya Zhang; Chenhui Ding; Rui Huang; Zhen Zhang; Jie Lv; Xiaowei Xie; Yuxi Chen; Yujing Li; Ying Sun; Yaofu Bai; Zhou Songyang; Wenbin Ma; Canquan Zhou; Junjiu Huang
Journal: Protein Cell Date: 2015-04-18 Impact factor: 14.870

10. Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors.

Authors: Jianbin Wang; Colin M Exline; Joshua J DeClercq; G Nicholas Llewellyn; Samuel B Hayward; Patrick Wai-Lun Li; David A Shivak; Richard T Surosky; Philip D Gregory; Michael C Holmes; Paula M Cannon
Journal: Nat Biotechnol Date: 2015-11-09 Impact factor: 54.908

127 in total

1. An effective vaginal gel to deliver CRISPR/Cas9 system encapsulated in poly (β-amino ester) nanoparticles for vaginal gene therapy.

Authors: Gang Niu; Zhuang Jin; Chong Zhang; Dan He; Xueqin Gao; Chenming Zou; Wei Zhang; Jiahui Ding; Bhudev C Das; Konstantin Severinov; Inga Isabel Hitzeroth; Priya Ranjan Debata; Xin Ma; Xun Tian; Qinglei Gao; Jun Wu; Zeshan You; Rui Tian; Zifeng Cui; Weiwen Fan; Weiling Xie; Zhaoyue Huang; Chen Cao; Wei Xu; Hongxian Xie; Hongyan Xu; Xiongzhi Tang; Yan Wang; Zhiying Yu; Hui Han; Songwei Tan; Shuqin Chen; Zheng Hu
Journal: EBioMedicine Date: 2020-07-22 Impact factor: 8.143

Delivery technologies for genome editing.

1. Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity.

2. RNAi-nanoparticulate manipulation of gene expression as a new functional genomics tool in the liver.

3. Rational design of a split-Cas9 enzyme complex.

4. Efficient delivery of genome-editing proteins using bioreducible lipid nanoparticles.

5. In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy.

6. Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells.

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

8. Autonomous zinc-finger nuclease pairs for targeted chromosomal deletion.

9. CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes.

10. Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors.

1. An effective vaginal gel to deliver CRISPR/Cas9 system encapsulated in poly (β-amino ester) nanoparticles for vaginal gene therapy.

Review 2. Manufacturing Cell Therapies Using Engineered Biomaterials.

Review 3. Non-viral delivery systems for CRISPR/Cas9-based genome editing: Challenges and opportunities.

4. Integrated design, execution, and analysis of arrayed and pooled CRISPR genome-editing experiments.

Review 5. Delivering the Messenger: Advances in Technologies for Therapeutic mRNA Delivery.

Review 6. Genome Editing with mRNA Encoding ZFN, TALEN, and Cas9.

7. Nontoxic nanopore electroporation for effective intracellular delivery of biological macromolecules.

8. Immunotherapy perspectives in the new era of B-cell editing.

9. Arbovirus Infections As Screening Tools for the Identification of Viral Immunomodulators and Host Antiviral Factors.

10. Nanoparticles for CRISPR-Cas9 delivery.