CRISPR technologies for genome, epigenome and transcriptome editing.

Autor: Villiger L; McGovern Institute for Brain Research, Massachusetts Institute of Technology Cambridge, Cambridge, MA, USA., Joung J; Whitehead Institute for Biomedical Research, Cambridge, MA, USA.; Department of Biology, Massachusetts Institute of Technology, Cambridge, MA, USA., Koblan L; Whitehead Institute for Biomedical Research, Cambridge, MA, USA.; Department of Biology, Massachusetts Institute of Technology, Cambridge, MA, USA., Weissman J; Whitehead Institute for Biomedical Research, Cambridge, MA, USA.; Department of Biology, Massachusetts Institute of Technology, Cambridge, MA, USA.; Howard Hughes Medical Institute, Massachusetts Institute of Technology, Cambridge, MA, USA.; David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, USA., Abudayyeh OO; McGovern Institute for Brain Research, Massachusetts Institute of Technology Cambridge, Cambridge, MA, USA. omar@abudayyeh.science., Gootenberg JS; McGovern Institute for Brain Research, Massachusetts Institute of Technology Cambridge, Cambridge, MA, USA. jgoot@mit.edu.
Jazyk: angličtina
Zdroj: Nature reviews. Molecular cell biology [Nat Rev Mol Cell Biol] 2024 Jun; Vol. 25 (6), pp. 464-487. Date of Electronic Publication: 2024 Feb 02.
DOI: 10.1038/s41580-023-00697-6
Abstrakt: Our ability to edit genomes lags behind our capacity to sequence them, but the growing understanding of CRISPR biology and its application to genome, epigenome and transcriptome engineering is narrowing this gap. In this Review, we discuss recent developments of various CRISPR-based systems that can transiently or permanently modify the genome and the transcriptome. The discovery of further CRISPR enzymes and systems through functional metagenomics has meaningfully broadened the applicability of CRISPR-based editing. Engineered Cas variants offer diverse capabilities such as base editing, prime editing, gene insertion and gene regulation, thereby providing a panoply of tools for the scientific community. We highlight the strengths and weaknesses of current CRISPR tools, considering their efficiency, precision, specificity, reliance on cellular DNA repair mechanisms and their applications in both fundamental biology and therapeutics. Finally, we discuss ongoing clinical trials that illustrate the potential impact of CRISPR systems on human health.
(© 2024. Springer Nature Limited.)
Databáze: MEDLINE
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