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Lentiviral CRISPR/Cas9-Mediated Genome Editing for the Study of Hematopoietic Cells in Disease Models
Published on: October 3, 2019
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Disease modeling and stem cell immunoengineering in regenerative medicine using CRISPR/Cas9 systems
Ainsley Mike Antao1, Janardhan Keshav Karapurkar1, Dong Ryul Lee2,3
1Graduate School of Biomedical Science and Engineering, Hanyang University, Seoul, South Korea.
Computational and Structural Biotechnology Journal
|December 11, 2020
Summary
CRISPR gene editing advances regenerative medicine by creating disease models and immune-engineered stem cells. This technology offers a realistic approach to overcoming transplantation barriers like HLA genes for universal donor cells.
Area of Science:
- Biotechnology
- Genetics
- Regenerative Medicine
Background:
- CRISPR/Cas systems are programmable nucleases driving progress in regenerative medicine.
- Disease models generated using CRISPR tools aid in understanding molecular aspects of diseases, including immune rejection.
- Major histocompatibility complex-human leukocyte antigen (HLA) genes impede advances in cell and tissue transplantation.
Purpose of the Study:
- To outline the structural and molecular frameworks of the type II CRISPR system.
- To present computational tools that aid in experimental design for CRISPR applications.
- To propose the generation of universal donor immune-engineered stem cells as a viable strategy in regenerative medicine.
Main Methods:
- Review of the structural and molecular frameworks of the type II CRISPR system.
- Description of computational tools for CRISPR experimental design.
- Hypothesis formulation based on CRISPR advancements and clinical trial progress.
Main Results:
- Detailed explanation of the type II CRISPR system's mechanisms.
- Introduction to computational resources for optimizing CRISPR experiments.
- Evidence supporting the feasibility of generating universal donor stem cells.
Conclusions:
- CRISPR technology is pivotal for advancing regenerative medicine.
- Immune-engineered stem cells offer a promising solution to HLA-mediated transplantation barriers.
- The development of universal donor cells via CRISPR is a realistic therapeutic strategy for various diseases.
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