Basic Principles and Clinical Applications of CRISPR-Based Genome Editing

Advances in sequencing technologies have facilitated the discovery of previously unknown genetic variants in both inherited and acquired disorders, and tools to correct these pathogenic variants are rapidly evolving. Since the first introduction of CRISPR-Cas9 in 2012, the field of CRISPR-based geno...

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Published inYonsei medical journal Vol. 63; no. 2; pp. 105 - 113
Main Authors Lim, Jung Min, Kim, Hyongbum Henry
Format Journal Article
LanguageEnglish
Published Korea (South) Yonsei University College of Medicine 01.02.2022
연세대학교의과대학
Subjects
Animals
CRISPR-Cas Systems - genetics
Gene Editing
Genome
Humans
Review
의학일반
CRISPR-Associated Protein 9
Genome editing
genetic therapy
gene editing
CRISPR-Cas systems
Online AccessGet full text
ISSN0513-5796
1976-2437
1976-2437
DOI10.3349/ymj.2022.63.2.105

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Abstract Advances in sequencing technologies have facilitated the discovery of previously unknown genetic variants in both inherited and acquired disorders, and tools to correct these pathogenic variants are rapidly evolving. Since the first introduction of CRISPR-Cas9 in 2012, the field of CRISPR-based genome editing has progressed immensely, giving hope to many patients suffering from genetic disorders that lack effective treatment. In this review, we will examine the basic principles of CRISPR-based genome editing, explain the mechanisms of new genome editors, including base editors and prime editors, and evaluate the therapeutic possibilities of CRISPR-based genome editing by focusing on recently published clinical trials and animal studies. Although efficacy and safety issues remain a large concern, we cannot deny that CRISPR-based genome editing will soon be prevalent in clinical practice.
AbstractList Advances in sequencing technologies have facilitated the discovery of previously unknown genetic variants in both inherited and acquired disorders, and tools to correct these pathogenic variants are rapidly evolving. Since the first introduction of CRISPR-Cas9 in 2012, the field of CRISPR-based genome editing has progressed immensely, giving hope to many patients suffering from genetic disorders that lack effective treatment. In this review, we will examine the basic principles of CRISPR-based genome editing, explain the mechanisms of new genome editors, including base editors and prime editors, and evaluate the therapeutic possibilities of CRISPR-based genome editing by focusing on recently published clinical trials and animal studies. Although efficacy and safety issues remain a large concern, we cannot deny that CRISPR-based genome editing will soon be prevalent in clinical practice.
Advances in sequencing technologies have facilitated the discovery of previously unknown genetic variants in both inherited andacquired disorders, and tools to correct these pathogenic variants are rapidly evolving. Since the first introduction of CRISPR-Cas9in 2012, the field of CRISPR-based genome editing has progressed immensely, giving hope to many patients suffering from geneticdisorders that lack effective treatment. In this review, we will examine the basic principles of CRISPR-based genome editing, explainthe mechanisms of new genome editors, including base editors and prime editors, and evaluate the therapeutic possibilitiesof CRISPR-based genome editing by focusing on recently published clinical trials and animal studies. Although efficacy and safetyissues remain a large concern, we cannot deny that CRISPR-based genome editing will soon be prevalent in clinical practice. KCI Citation Count: 0
Advances in sequencing technologies have facilitated the discovery of previously unknown genetic variants in both inherited and acquired disorders, and tools to correct these pathogenic variants are rapidly evolving. Since the first introduction of CRISPR-Cas9 in 2012, the field of CRISPR-based genome editing has progressed immensely, giving hope to many patients suffering from genetic disorders that lack effective treatment. In this review, we will examine the basic principles of CRISPR-based genome editing, explain the mechanisms of new genome editors, including base editors and prime editors, and evaluate the therapeutic possibilities of CRISPR-based genome editing by focusing on recently published clinical trials and animal studies. Although efficacy and safety issues remain a large concern, we cannot deny that CRISPR-based genome editing will soon be prevalent in clinical practice.Advances in sequencing technologies have facilitated the discovery of previously unknown genetic variants in both inherited and acquired disorders, and tools to correct these pathogenic variants are rapidly evolving. Since the first introduction of CRISPR-Cas9 in 2012, the field of CRISPR-based genome editing has progressed immensely, giving hope to many patients suffering from genetic disorders that lack effective treatment. In this review, we will examine the basic principles of CRISPR-based genome editing, explain the mechanisms of new genome editors, including base editors and prime editors, and evaluate the therapeutic possibilities of CRISPR-based genome editing by focusing on recently published clinical trials and animal studies. Although efficacy and safety issues remain a large concern, we cannot deny that CRISPR-based genome editing will soon be prevalent in clinical practice.
Author Kim, Hyongbum Henry
Lim, Jung Min
AuthorAffiliation 4 Institute for Immunology and Immunological Diseases, Yonsei University College of Medicine, Seoul, Korea
7 Graduate Program of Nano Biomedical Engineering (NanoBME), Advanced Science Institute, Yonsei University, Seoul, Korea
2 Brain Korea 21 Plus Project for Medical Sciences, Yonsei University College of Medicine, Seoul, Korea
6 Center for Nanomedicine, Institute for Basic Science (IBS), Seoul, Korea
3 Severance Biomedical Science Institute, Yonsei University College of Medicine, Seoul, Korea
1 Department of Pharmacology, Yonsei University College of Medicine, Seoul, Korea
5 Graduate Program of NanoScience and Technology, Yonsei University, Seoul, Korea
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– name: 5 Graduate Program of NanoScience and Technology, Yonsei University, Seoul, Korea
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Issue 2
Keywords CRISPR-Associated Protein 9
Genome editing
genetic therapy
gene editing
CRISPR-Cas systems
Language English
License Copyright: Yonsei University College of Medicine 2022.
This is an Open Access article distributed under the terms of the Creative Commons Attribution Non-Commercial License (https://creativecommons.org/licenses/by-nc/4.0/) which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited.
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Snippet Advances in sequencing technologies have facilitated the discovery of previously unknown genetic variants in both inherited and acquired disorders, and tools...
Advances in sequencing technologies have facilitated the discovery of previously unknown genetic variants in both inherited andacquired disorders, and tools to...
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SubjectTerms Animals
CRISPR-Cas Systems - genetics
Gene Editing
Genome
Humans
Review
의학일반
Title Basic Principles and Clinical Applications of CRISPR-Based Genome Editing
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