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拓展基因编辑工具和靶点。

Expanding the Toolbox and Targets for Gene Editing.

机构信息

Gene Expression Laboratory, Salk Institute for Biological Studies, La Jolla, CA 92037, USA.

Gene Expression Laboratory, Salk Institute for Biological Studies, La Jolla, CA 92037, USA.

出版信息

Trends Mol Med. 2021 Mar;27(3):203-206. doi: 10.1016/j.molmed.2020.12.005. Epub 2021 Jan 21.


DOI:10.1016/j.molmed.2020.12.005
PMID:33487569
Abstract

Genome editing holds great promise for treating a range of human genetic diseases. While emerging clustered regularly interspaced short-palindromic repeats (CRISPR) technologies allow editing of the nuclear genome, it is still not possible to precisely manipulate mitochondrial DNA (mtDNA). Here, we summarize past developments and recent advances in nuclear and mitochondrial genome editing.

摘要

基因组编辑在治疗一系列人类遗传疾病方面具有巨大的潜力。虽然新兴的成簇规律间隔短回文重复序列(CRISPR)技术允许对核基因组进行编辑,但仍然不可能精确地操作线粒体 DNA(mtDNA)。在这里,我们总结了核基因组和线粒体基因组编辑的过去发展和最新进展。

相似文献

[1]
Expanding the Toolbox and Targets for Gene Editing.

Trends Mol Med. 2021-3

[2]
The Future of Genome Editing.

Cell. 2018-5-31

[3]
CRISPR-cas9 genome editing delivery systems for targeted cancer therapy.

Life Sci. 2021-2-15

[4]
Gene Editing With CRISPR/Cas9 RNA-Directed Nuclease.

Circ Res. 2017-3-3

[5]
Development and application of CRISPR/Cas9 technologies in genomic editing.

Hum Mol Genet. 2018-8-1

[6]
The Implications of CRISPR-Cas9 Genome Editing for IR.

J Vasc Interv Radiol. 2018-9

[7]
Therapeutic and diagnostic relevance of Crispr technology.

Biomed Pharmacother. 2021-6

[8]
CRISPR/Cas9 System and its Research Progress in Gene Therapy.

Anticancer Agents Med Chem. 2019

[9]
CRISPR Advancements for Human Health.

Mo Med. 2024

[10]
[Advances in application of clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated 9 system in stem cells research].

Zhonghua Shao Shang Za Zhi. 2018-4-20

引用本文的文献

[1]
Gene therapy for genetic diseases: challenges and future directions.

MedComm (2020). 2025-2-13

[2]
Emerging Strategies to Overcome Current CAR-T Therapy Dilemmas - Exosomes Derived from CAR-T Cells.

Int J Nanomedicine. 2024

[3]
Therapeutic In Vivo Gene Editing Achieved by a Hypercompact CRISPR-Cas12f1 System Delivered with All-in-One Adeno-Associated Virus.

Adv Sci (Weinh). 2024-5

[4]
Exploring Molecular Targets for Mitochondrial Therapies in Neurodegenerative Diseases.

Int J Mol Sci. 2023-8-6

[5]
Potential of Mitochondrial Genome Editing for Human Fertility Health.

Front Genet. 2021-7-20

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