Engineered miniature CRISPR-Cas system for mammalian genome regulation and editing.

  title={Engineered miniature CRISPR-Cas system for mammalian genome regulation and editing.},
  author={Xiaoshu Xu and Augustine Chemparathy and Leiping Zeng and Hannah R. Kempton and Stephen Shang and Muneaki Nakamura and Lei S. Qi},
  journal={Molecular cell},

Engineered Cas12i2 is a versatile high-efficiency platform for therapeutic genome editing

An engineered Type V-I CRISPR system (Cas12i), ABR-001, which utilizes a tracr-less guide RNA and is established as a versatile, specific, and high-performance platform for ex vivo and in vivo gene therapy.

Synergistic engineering of CRISPR-Cas nucleases enables robust mammalian genome editing

Multiplexed genome regulation in vivo with hyper-efficient Cas12a

A hyper-efficient Lachnospiraceae bacterium Cas12a variant with enhanced editing efficiency and its catalytically dead version hyperdCas12a showing significantly enhanced efficacy for gene activation, particularly at low concentrations of crRNA is developed.

Tools and targets: The dual role of plant viruses in CRISPR-Cas genome editing.

The utility of CRISPR-Cas technology to enhance biotic resistance with a special focus on plant virus diseases is explored and the challenges and potential that VIGE holds in future breeding technologies are discussed.

CRISPR based therapeutics: a new paradigm in cancer precision medicine

The latest developments in the field in terms of optimization of performance of the CRISPR-Cas elements should fuel greater application of the latter in the realm of precision medicine.

An engineered xCas12i with high activity, high specificity and broad PAM range

This study provides highly efficient and specific tools for gene therapy by engineering the PAM-interacting, REC, and RuvC domains for enhanced cleavage activity and specificity in Cas12i.

The use of new CRISPR tools in cardiovascular research and medicine

How these novel CRISPR tools are used to investigate biological processes and disease pathophysiology for cardiovascular research and medicine and the prospect of therapeutic genome editing by CRISpr tools to cure genetic cardiovascular diseases are described.

Non-GM Genome Editing Approaches in Crops

The development of transgene-free genome editing technologies can address many problems associated with transgenic-based approaches and the use of viral vectors for delivery of CRISPR/Cas components has recently emerged as a powerful alternative but it requires further exploration.



A CRISPR-dCas Toolbox for Genetic Engineering and Synthetic Biology.

Engineering of CRISPR-Cas12b for human genome editing

This work establishes a third RNA-guided nuclease platform, in addition to Cas9 and Cpf1/Cas12a, for genome editing in human cells, and identifies a promising candidate for human gene editing from Bacillus hisashii, BhCas12b.

Programmed DNA destruction by miniature CRISPR-Cas14 enzymes

Metagenomic data show that multiple CRISPR-Cas14 systems evolved independently and suggest a potential evolutionary origin of single-effector CRISpr-based adaptive immunity, as well as a fast and high-fidelity nucleic acid detection system that enabled detection ofsingle-nucleotide polymorphisms.

Multiplex Genome Engineering Using CRISPR/Cas Systems

Two different type II CRISPR/Cas systems are engineered and it is demonstrated that Cas9 nucleases can be directed by short RNAs to induce precise cleavage at endogenous genomic loci in human and mouse cells, demonstrating easy programmability and wide applicability of the RNA-guided nuclease technology.

Development of CRISPR-Cas systems for genome editing and beyond

  • F. Zhang
  • Biology
    Quarterly Reviews of Biophysics
  • 2019
A personal perspective on the development of CRISPR-Cas9 for genome editing within the broader context of the field is provided and the work to discover novel Cas effectors and develop them into additional molecular tools is discussed.

CRISPR-CasΦ from huge phages is a hypercompact genome editor

A minimal functional CRISpr-Cas system, comprising a single ~70-kilodalton protein, CasΦ, and a CRISPR array, encoded exclusively in the genomes of huge bacteriophages, which provides a hypercompact addition to the genome-editing toolbox.

Cornerstones of CRISPR–Cas in drug discovery and therapy

How CRISPR–Cas can affect the next generation of drugs by accelerating the identification and validation of high-value targets, uncovering high-confidence biomarkers and developing differentiated breakthrough therapies is discussed.

Genome-scale transcriptional activation by an engineered CRISPR-Cas9 complex

Structural-guided engineering of a CRISPR-Cas9 complex to mediate efficient transcriptional activation at endogenous genomic loci is described and the potential of Cas9-based activators as a powerful genetic perturbation technology is demonstrated.