Clinical trials and promising preclinical applications of CRISPR/Cas gene editing

被引：11

作者：

Cerci, Baris ^{[1
]}

Uzay, Ihsan Alp ^{[1
]}

Kara, Mustafa Kemal ^{[1
]}

Dincer, Pervin ^{[2
]}

机构：

[1] Hacettepe Univ, Fac Med, TR-06100 Ankara, Turkiye

[2] Hacettepe Univ, Dept Med Biol, Fac Med, TR-06100 Ankara, Turkiye

来源：

LIFE SCIENCES | 2023年 / 312卷

关键词：

CRISPR-Cas; Gene editing; Rare diseases; Preclinical application; Clinical trials; ORNITHINE TRANSCARBAMYLASE DEFICIENCY; AMYOTROPHIC-LATERAL-SCLEROSIS; CHRONIC GRANULOMATOUS-DISEASE; METABOLIC LIVER-DISEASE; ACTIVATED KILLER-CELLS; STRAND BREAK REPAIR; MUSCULAR-DYSTROPHY; T-CELLS; ALPHA-1-ANTITRYPSIN DEFICIENCY; HOMOLOGOUS RECOMBINATION;

D O I：

10.1016/j.lfs.2022.121204

中图分类号：

R-3 [医学研究方法]; R3 [基础医学];

学科分类号：

1001 ;

摘要：

Treatment of genetic disorders by genomic manipulation has been the unreachable goal of researchers for many decades. Although our understanding of the genetic basis of genetic diseases has advanced tremendously in the last few decades, the tools developed for genomic editing were not efficient and practical for their use in the clinical setting until now. The recent advancements in the research of Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and CRISPR-associated protein (Cas) systems offered an easy and efficient way to edit the genome and accelerated the research on their potential use in the treatment of genetic disorders. In this review, we summarize the clinical trials that evaluate the CRISPR/Cas systems for treating different genetic diseases and highlight promising preclinical research on CRISPR/Cas mediated treatment of a great diversity of genetic disorders. Ultimately, we discuss the future of CRISPR/Cas mediated genome editing in genetic diseases.

引用

页数：22

共 50 条

[21] Current applications and future perspective of CRISPR/Cas9 gene editing in cancer
Wang, Si-Wei
Gao, Chao
Zheng, Yi-Min
Yi, Li
Lu, Jia-Cheng
Huang, Xiao-Yong
Cai, Jia-Bin
Zhang, Peng-Fei
Cui, Yue-Hong
Ke, Ai-Wu
MOLECULAR CANCER, 2022, 21 (01)
[22] CRISPR/Cas9 Genome Editing Tool: A Promising Tool for Therapeutic Applications on Respiratory Diseases
Shaikh, Sadiya Bi
Bhandary, Yashodhar Prabhakar
CURRENT GENE THERAPY, 2020, 20 (05) : 333 - 346
[23] CRISPR/Cas9 gene editing in a chicken model: current approaches and applications
Luiza Chojnacka-Puchta
Dorota Sawicka
Journal of Applied Genetics, 2020, 61 : 221 - 229
[24] Viral Delivery of Compact CRISPR-Cas12f for Gene Editing Applications
Sharrar, Allison
Meacham, Zuriah
Staples-Ager, Johanna
de Tacca, Luisa Arake
Rabuka, David
Collingwood, Trevor
Schelle, Michael
CRISPR JOURNAL, 2024, 7 (03): : 150 - 155
[25] Emerging CRISPR/Cas9 applications for T-cell gene editing
Preece, Roland
Georgiadis, Christos
EMERGING TOPICS IN LIFE SCIENCES, 2019, 3 (03) : 261 - 275
[26] Controlling CRISPR-Cas9 Gene Editing
Dowdy, Steven F.
NEW ENGLAND JOURNAL OF MEDICINE, 2019, 381 (03): : 289 - 290
[27] Advancing CRISPR/Cas gene editing with machine learning
Fong, John H. C.
Wong, Alan S. L.
CURRENT OPINION IN BIOMEDICAL ENGINEERING, 2023, 28
[28] CRISPR-Cas Gene Editing to the Genetic Rescue
Goetzl, Edward J.
Alpert, Joseph S.
AMERICAN JOURNAL OF MEDICINE, 2024, 137 (05): : 386 - 389
[29] Introduction to CRISPR-Cas9 Gene Editing
Mason, J. M.
BIRTH DEFECTS RESEARCH, 2020, 112 (11): : 809 - 809
[30] The CRISPR-Cas toolbox and gene editing technologies
Liu, Guanwen
Lin, Qiupeng
Jin, Shuai
Gao, Caixia
MOLECULAR CELL, 2022, 82 (02) : 333 - 347

← 1 2 3 4 5 →