Related Experiment Video
Updated: Sep 9, 2025

09:37
Genome Editing and Directed Differentiation of hPSCs for Interrogating Lineage Determinants in Human Pancreatic Development
Published on: March 5, 2017
13.2K
The Interface of Gene Editing with Regenerative Medicine
Veronica E Farag1, Elsie A Devey1, Kam W Leong1,2
1Department of Biomedical Engineering, Columbia University, New York City, NY 10027, USA.
Summary
Genome editing with CRISPR technologies offers precise alternatives for regenerative medicine, advancing treatments for genetic diseases and tissue repair. This review highlights CRISPR
Area of Science:
- Regenerative Medicine
- Genome Engineering
- Gene Editing Technologies
Background:
- Regenerative medicine aims to repair tissues and restore function, with existing therapies approved for wound healing and orthopedic injuries.
- Current strategies utilize progenitor cells and biological stimuli but face challenges like off-target effects and lack of precision.
- The field is evolving towards more precise and efficient genome engineering technologies.
Purpose of the Study:
- To review the progress and future prospects of CRISPR technologies in regenerative medicine.
- To explore how gene editing advances therapeutic applications and aids research in tissue development and disease progression.
Main Methods:
- Review of CRISPR-based gene editing systems.
- Analysis of CRISPR applications in regenerative medicine and tissue engineering.
- Exploration of CRISPR's role as a research tool.
Main Results:
- CRISPR enables precise gene editing, including knock-ins, knockouts, transcriptional modulation, and base conversions.
- CRISPR facilitates the treatment of genetic and degenerative diseases.
- CRISPR aids in controlling cell fate for tissue repair and enhancing tissue function.
Conclusions:
- CRISPR technologies represent a significant advancement in regenerative medicine, offering precise and versatile tools.
- Gene editing with CRISPR is transforming therapeutic applications and deepening our understanding of biological processes.
- The integration of CRISPR promises to overcome limitations of traditional regenerative medicine approaches.
Related Concept Videos
Gene Therapy
25.8K
Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
25.8K
What is Genetic Engineering?
75.4K
Overview
75.4K
CRISPR
52.9K
Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...
52.9K
CRISPR/Cas9 Genome Editing
211
The CRISPR-Cas system serves as a bacterial defense mechanism against invading genetic elements such as viruses and plasmids, forming the foundation for its adaptation as a powerful genome-editing tool. Originally discovered in prokaryotes, this system has been repurposed to revolutionize genetic engineering across a wide range of organisms, including plants, animals, and humans. The core component, Cas9, is an endonuclease derived from Streptococcus pyogenes, capable of introducing...
211
In-vitro Mutagenesis
14.2K
To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.
14.2K
Stem Cell Therapy for Tissue Regeneration
4.2K
Stem cell therapy is a method used in regenerative medicine to repair and restore function to damaged tissues and organs. Stem cells have the potential to proliferate and differentiate into various tissue types, making them ideal candidates for tissue regeneration. For example, hematopoietic stem cell transplants are commonly used in blood cancer treatment to replenish damaged bone marrow and restore healthy blood cells.
Types of Stem Cells used in Stem Cell Therapy
The two main cell...
Types of Stem Cells used in Stem Cell Therapy
The two main cell...
4.2K

