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Genome Editing

Recent advances in genome editing technologies have substantially improved our ability to make precise changes in the genomes of eukaryotic cells. Programmable nucleases, particularly the CRISPR/Cas system, are already revolutionizing our ability to interrogate the function of the genome and can potentially be used clinically to correct or introduce genetic mutations to treat diseases that are refractory to traditional therapies. This collection of articles from the Nature Research journals provides an overview of current progress in developing targeted genome editing technologies. A selection of protocols for using and adapting these tools in your own lab is also included.

News and comments

Research

  • Nature Communications | Article | open

    Cas9 and Cpf1 have both been adapted for genome engineering, editing and gene expression regulation. Here the authors design a fusion guide RNA that can interact with both proteins for multiple and orthogonal genome manipulation.

    • Jiyeon Kweon
    • , An-Hee Jang
    • , Da-eun Kim
    • , Jin Wook Yang
    • , Mijung Yoon
    • , Ha Rim Shin
    • , Jin-Soo Kim
    •  &  Yongsub Kim
  • Nature | Letter

    CRISPR–Cas9 genome editing is used to correct a dominant-negative mutation in a mouse model of inherited deafness, resulting in improvements in cochlear function and hearing.

    • Xue Gao
    • , Yong Tao
    • , Veronica Lamas
    • , Mingqian Huang
    • , Wei-Hsi Yeh
    • , Bifeng Pan
    • , Yu-Juan Hu
    • , Johnny H. Hu
    • , David B. Thompson
    • , Yilai Shu
    • , Yamin Li
    • , Hongyang Wang
    • , Shiming Yang
    • , Qiaobing Xu
    • , Daniel B. Polley
    • , M. Charles Liberman
    • , Wei-Jia Kong
    • , Jeffrey R. Holt
    • , Zheng-Yi Chen
    •  &  David R. Liu
  • Nature Communications | Article | open

    RNA regulatory elements (RREs) are important post-transcriptional control features but studying them requires disrupting their activity without disturbing cellular homeostasis. Here the authors present GenERA, a CRISPR-Cas9 screening platform of in situ analysis of native RREs.

    • Qianxin Wu
    • , Quentin R. V. Ferry
    • , Toni A. Baeumler
    • , Yale S. Michaels
    • , Dimitrios M. Vitsios
    • , Omer Habib
    • , Roland Arnold
    • , Xiaowei Jiang
    • , Stefano Maio
    • , Bruno R. Steinkraus
    • , Marta Tapia
    • , Paolo Piazza
    • , Ni Xu
    • , Georg A. Holländer
    • , Thomas A. Milne
    • , Jin-Soo Kim
    • , Anton J. Enright
    • , Andrew R. Bassett
    •  &  Tudor A. Fulga
  • Nature Communications | Article | open

    G-protein-coupled receptors are a large and diverse group of eukaryotic membrane receptors. Here the authors couple GPCRs to dCas9 to link extracellular sensing to genome regulation.

    • Nathan H. Kipniss
    • , P. C. Dave P. Dingal
    • , Timothy R. Abbott
    • , Yuchen Gao
    • , Haifeng Wang
    • , Antonia A. Dominguez
    • , Louai Labanieh
    •  &  Lei S. Qi
  • Nature Communications | Article | open

    Cpf1 is a promising addition to the CRISPR toolkit but displays wide variability of activity in different eurkaryotes. Here the authors identify temperature as a modulator of activity and use this to efficiently edit ectothermic vertebrate species.

    • Miguel A. Moreno-Mateos
    • , Juan P. Fernandez
    • , Romain Rouet
    • , Charles E. Vejnar
    • , Maura A. Lane
    • , Emily Mis
    • , Mustafa K. Khokha
    • , Jennifer A. Doudna
    •  &  Antonio J. Giraldez
  • Nature Biotechnology | Article

    Efficient Cas9 genome editing in vivo is achieved without viral vectors using chemically modified single-guide RNAs.

    • Hao Yin
    • , Chun-Qing Song
    • , Sneha Suresh
    • , Qiongqiong Wu
    • , Stephen Walsh
    • , Luke Hyunsik Rhym
    • , Esther Mintzer
    • , Mehmet Fatih Bolukbasi
    • , Lihua Julie Zhu
    • , Kevin Kauffman
    • , Haiwei Mou
    • , Alicia Oberholzer
    • , Junmei Ding
    • , Suet-Yan Kwan
    • , Roman L Bogorad
    • , Timofei Zatsepin
    • , Victor Koteliansky
    • , Scot A Wolfe
    • , Wen Xue
    • , Robert Langer
    •  &  Daniel G Anderson

Reviews

  • Nature Medicine | Review Article

    In this Review, Cathomen and colleagues present the latest advances, including improvements in nuclease specificity and delivery, that will expedite the clinical translation of genome editing.

    • Tatjana I Cornu
    • , Claudio Mussolino
    •  &  Toni Cathomen
  • Nature Reviews Molecular Cell Biology | Review Article

    The CRISPR–Cas9 system is a powerful, sequence-specific tool that was initially developed for gene and genome editing. The recent adoption of nuclease-deactivated Cas9 (dCas9) has enabled expansion of the use of the system to multiplexed and inducible transcription regulation, genome-wide screens and cell fate engineering.

    • Antonia A. Dominguez
    • , Wendell A. Lim
    •  &  Lei S. Qi
  • Nature Reviews Drug Discovery | Review Article

    Genome editing has emerged as an attractive approach to therapeutically manipulate gene expression. Here, Anderson and colleagues provide an overview of genome-editing platforms, focusing on the methods and challenges of intracellular biomacromolecule delivery. Preclinical and clinical trials involving genome-editing technologies are also discussed.

    • Hao Yin
    • , Kevin J. Kauffman
    •  &  Daniel G. Anderson

Protocols