CRISPR-mediated genome editing and human diseases
CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) technology has emerged as a powerful technology for genome editing and is now widely used in basic biomedical research to explore gene function. More recently, this technology has been increasingly applied to the study or treatment o...
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doaj-5d227910294f418db065169bf02006e82020-11-25T00:55:47ZengElsevierGenes and Diseases2352-30422016-12-013424425110.1016/j.gendis.2016.07.003CRISPR-mediated genome editing and human diseasesLiquan Cai0Alfred L. Fisher1Haochu Huang2Zijian Xie3Marshall Institute for Interdisciplinary Research (MIIR), Marshall University, Weisberg Engineering Complex, Marshall University, 1628 Third Avenue, Huntington, WV 25703, USAUniversity of Texas Health Science Center at San Antonio, Department of Medicine, Division of Geriatrics, Gerontology, and Palliative Medicine, San Antonio, TX 78229, USACommittee on Immunology, Section of Rheumatology, Department of Medicine, Knapp Center for Lupus and Immunology Research, University of Chicago, Chicago, IL 60637, USAMarshall Institute for Interdisciplinary Research (MIIR), Marshall University, Weisberg Engineering Complex, Marshall University, 1628 Third Avenue, Huntington, WV 25703, USACRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) technology has emerged as a powerful technology for genome editing and is now widely used in basic biomedical research to explore gene function. More recently, this technology has been increasingly applied to the study or treatment of human diseases, including Barth syndrome effects on the heart, Duchenne muscular dystrophy, hemophilia, β-Thalassemia, and cystic fibrosis. CRISPR/Cas9 (CRISPR-associated protein 9) genome editing has been used to correct disease-causing DNA mutations ranging from a single base pair to large deletions in model systems ranging from cells in vitro to animals in vivo. In addition to genetic diseases, CRISPR/Cas9 gene editing has also been applied in immunology-focused applications such as the targeting of C-C chemokine receptor type 5, the programmed death 1 gene, or the creation of chimeric antigen receptors in T cells for purposes such as the treatment of the acquired immune deficiency syndrome (AIDS) or promoting anti-tumor immunotherapy. Furthermore, this technology has been applied to the genetic manipulation of domesticated animals with the goal of producing biologic medical materials, including molecules, cells or organs, on a large scale. Finally, CRISPR/Cas9 has been teamed with induced pluripotent stem (iPS) cells to perform multiple tissue engineering tasks including the creation of disease models or the preparation of donor-specific tissues for transplantation. This review will explore the ways in which the use of CRISPR/Cas9 is opening new doors to the treatment of human diseases.http://www.sciencedirect.com/science/article/pii/S2352304216300356CRISPRDNA double-stranded breakGenome editingHuman diseasesiPS cells |
collection |
DOAJ |
language |
English |
format |
Article |
sources |
DOAJ |
author |
Liquan Cai Alfred L. Fisher Haochu Huang Zijian Xie |
spellingShingle |
Liquan Cai Alfred L. Fisher Haochu Huang Zijian Xie CRISPR-mediated genome editing and human diseases Genes and Diseases CRISPR DNA double-stranded break Genome editing Human diseases iPS cells |
author_facet |
Liquan Cai Alfred L. Fisher Haochu Huang Zijian Xie |
author_sort |
Liquan Cai |
title |
CRISPR-mediated genome editing and human diseases |
title_short |
CRISPR-mediated genome editing and human diseases |
title_full |
CRISPR-mediated genome editing and human diseases |
title_fullStr |
CRISPR-mediated genome editing and human diseases |
title_full_unstemmed |
CRISPR-mediated genome editing and human diseases |
title_sort |
crispr-mediated genome editing and human diseases |
publisher |
Elsevier |
series |
Genes and Diseases |
issn |
2352-3042 |
publishDate |
2016-12-01 |
description |
CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) technology has emerged as a powerful technology for genome editing and is now widely used in basic biomedical research to explore gene function. More recently, this technology has been increasingly applied to the study or treatment of human diseases, including Barth syndrome effects on the heart, Duchenne muscular dystrophy, hemophilia, β-Thalassemia, and cystic fibrosis. CRISPR/Cas9 (CRISPR-associated protein 9) genome editing has been used to correct disease-causing DNA mutations ranging from a single base pair to large deletions in model systems ranging from cells in vitro to animals in vivo. In addition to genetic diseases, CRISPR/Cas9 gene editing has also been applied in immunology-focused applications such as the targeting of C-C chemokine receptor type 5, the programmed death 1 gene, or the creation of chimeric antigen receptors in T cells for purposes such as the treatment of the acquired immune deficiency syndrome (AIDS) or promoting anti-tumor immunotherapy. Furthermore, this technology has been applied to the genetic manipulation of domesticated animals with the goal of producing biologic medical materials, including molecules, cells or organs, on a large scale. Finally, CRISPR/Cas9 has been teamed with induced pluripotent stem (iPS) cells to perform multiple tissue engineering tasks including the creation of disease models or the preparation of donor-specific tissues for transplantation. This review will explore the ways in which the use of CRISPR/Cas9 is opening new doors to the treatment of human diseases. |
topic |
CRISPR DNA double-stranded break Genome editing Human diseases iPS cells |
url |
http://www.sciencedirect.com/science/article/pii/S2352304216300356 |
work_keys_str_mv |
AT liquancai crisprmediatedgenomeeditingandhumandiseases AT alfredlfisher crisprmediatedgenomeeditingandhumandiseases AT haochuhuang crisprmediatedgenomeeditingandhumandiseases AT zijianxie crisprmediatedgenomeeditingandhumandiseases |
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