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Literature DB >> 26121415

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

Ayal Hendel¹, Rasmus O Bak¹, Joseph T Clark¹, Andrew B Kennedy², Daniel E Ryan², Subhadeep Roy³, Israel Steinfeld⁴, Benjamin D Lunstad³, Robert J Kaiser², Alec B Wilkens¹, Rosa Bacchetta¹, Anya Tsalenko², Douglas Dellinger³, Laurakay Bruhn², Matthew H Porteus¹.

Abstract

CRISPR-Cas-mediated genome editing relies on guide RNAs that direct site-specific DNA cleavage facilitated by the Cas endonuclease. Here we report that chemical alterations to synthesized single guide RNAs (sgRNAs) enhance genome editing efficiency in human primary T cells and CD34(+) hematopoietic stem and progenitor cells. Co-delivering chemically modified sgRNAs with Cas9 mRNA or protein is an efficient RNA- or ribonucleoprotein (RNP)-based delivery method for the CRISPR-Cas system, without the toxicity associated with DNA delivery. This approach is a simple and effective way to streamline the development of genome editing with the potential to accelerate a wide array of biotechnological and therapeutic applications of the CRISPR-Cas technology.

Entities: CellLine Chemical Disease Gene Species

Mesh：

Substances：
RNA, Guide

Year: 2015 PMID： 26121415 PMCID： PMC4729442 DOI： 10.1038/nbt.3290

Source DB: PubMed Journal: Nat Biotechnol ISSN： 1087-0156 Impact factor: 54.908

24 in total

1. Streamlined process for the chemical synthesis of RNA using 2'-O-thionocarbamate-protected nucleoside phosphoramidites in the solid phase.

Authors: Douglas J Dellinger; Zoltán Timár; Joel Myerson; Agnieszka B Sierzchala; John Turner; Fernando Ferreira; Zoltán Kupihár; Geraldine Dellinger; Kenneth W Hill; James A Powell; Jeffrey R Sampson; Marvin H Caruthers
Journal: J Am Chem Soc Date: 2011-07-11 Impact factor: 15.419

2. Efficient ablation of genes in human hematopoietic stem and effector cells using CRISPR/Cas9.

Authors: Pankaj K Mandal; Leonardo M R Ferreira; Ryan Collins; Torsten B Meissner; Christian L Boutwell; Max Friesen; Vladimir Vrbanac; Brian S Garrison; Alexei Stortchevoi; David Bryder; Kiran Musunuru; Harrison Brand; Andrew M Tager; Todd M Allen; Michael E Talkowski; Derrick J Rossi; Chad A Cowan
Journal: Cell Stem Cell Date: 2014-11-06 Impact factor: 24.633

3. Site-specific integration and tailoring of cassette design for sustainable gene transfer.

Authors: Angelo Lombardo; Daniela Cesana; Pietro Genovese; Bruno Di Stefano; Elena Provasi; Daniele F Colombo; Margherita Neri; Zulma Magnani; Alessio Cantore; Pietro Lo Riso; Martina Damo; Oscar M Pello; Michael C Holmes; Philip D Gregory; Angela Gritti; Vania Broccoli; Chiara Bonini; Luigi Naldini
Journal: Nat Methods Date: 2011-08-21 Impact factor: 28.547

4. 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

10. Clonal expansion and myeloid leukemia progression modeled by multiplex gene editing of murine hematopoietic progenitor cells.

Authors: Xiangguo Shi; Ayumi Kitano; Yajian Jiang; Victor Luu; Kevin A Hoegenauer; Daisuke Nakada
Journal: Exp Hematol Date: 2018-05-08 Impact factor: 3.084

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

1. Streamlined process for the chemical synthesis of RNA using 2'-O-thionocarbamate-protected nucleoside phosphoramidites in the solid phase.

2. Efficient ablation of genes in human hematopoietic stem and effector cells using CRISPR/Cas9.

3. Site-specific integration and tailoring of cassette design for sustainable gene transfer.

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

5. Programmable RNA recognition and cleavage by CRISPR/Cas9.

6. Fast and accurate long-read alignment with Burrows-Wheeler transform.

7. Dynamic imaging of genomic loci in living human cells by an optimized CRISPR/Cas system.

8. CRISPR/Cas9 systems targeting β-globin and CCR5 genes have substantial off-target activity.

9. COSMID: A Web-based Tool for Identifying and Validating CRISPR/Cas Off-target Sites.

10. Easy quantitative assessment of genome editing by sequence trace decomposition.

1. Synthetic CRISPR RNA-Cas9-guided genome editing in human cells.

Review 2. Combining CRISPR/Cas9 and rAAV Templates for Efficient Gene Editing.

Review 3. Creating and evaluating accurate CRISPR-Cas9 scalpels for genomic surgery.

4. Stepping toward therapeutic CRISPR.

Review 5. Salient Features of Endonuclease Platforms for Therapeutic Genome Editing.

6. Design and assessment of engineered CRISPR-Cpf1 and its use for genome editing.

7. Spatiotemporal Control of CRISPR/Cas9 Function in Cells and Zebrafish using Light-Activated Guide RNA.

8. Concurrent Hydrogenation of Three Functional Groups Enables Synthesis of C3'-Homologated Nucleoside Amino Acids.

9. CRISPR-Mediated Integration of Large Gene Cassettes Using AAV Donor Vectors.

10. Clonal expansion and myeloid leukemia progression modeled by multiplex gene editing of murine hematopoietic progenitor cells.