Theoretical and Natural Science
- The Open Access Proceedings Series for Conferences
Theoretical and Natural Science
Vol. 23, 20 December 2023
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Access | Article
CRISPR-based genomic modification method in the treatment of hematological diseases
* Author to whom correspondence should be addressed.
Abstract
Mutations in hematopoietic stem cells are the main cause of most blood diseases. Hematopoietic stem cell transplantation, which is the main treatment for hematopoietic stem cell disorders ,brings many negative effects according to the clinical cases, such as high toxic side effects, limited donor source, high recurrence rate, and high treatment cost .CRISPR/Cas acts as an immune system in prokaryote that grants them an ability to resist invasion by exogenous mobile genetic elements (MGE), which is a new generation of gene editing technology after the appearance of TALEN and ZFN. The immune system functions in three steps: adaptation, expression, and interference. It enables genomic modification by unwinding the double strands of DNA and inducing DNA repair processes such as homologydirected repair (HDR) and/or non-homologous end joining (NHEJ). So, this paper discusses the utilization of Crispr system in the treatment of β-hemoglobinopathy, Chronic myeloid leukemia (CML) , Hemophilia and summarizes the current limitations of this technology, such as the editing efficiency, potential immunogenicity of editing tools and off-target effects , while it still remains promising as to its future prospects for research. This technology is very likely to replace traditional therapies and achieve a complete cure for the blood disease.
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Data Availability
The datasets used and/or analyzed during the current study will be available from the authors upon reasonable request.
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- Volume Title
- Proceedings of the 3rd International Conference on Biological Engineering and Medical Science
- ISBN (Print)
- 978-1-83558-219-0
- ISBN (Online)
- 978-1-83558-220-6
- Published Date
- 20 December 2023
- Series
- Theoretical and Natural Science
- ISSN (Print)
- 2753-8818
- ISSN (Online)
- 2753-8826
- DOI
- 10.54254/2753-8818/23/20231026
- Copyright
- 20 December 2023
- Open Access
- This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited