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CRISPR

CRISPR

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...

CRISPR and crRNAs

CRISPR and crRNAs

Bacteria and archaea are susceptible to viral infections just like eukaryotes; therefore, they have developed a unique adaptive immune system to protect themselves. Clustered regularly interspaced short palindromic repeats and CRISPR-associated proteins (CRISPR-Cas) are present in more than 45% of known bacteria and 90% of known archaea.
The CRISPR-Cas system stores a copy of foreign DNA in the host genome and uses it to identify the foreign DNA upon reinfection. CRISPR-Cas has three different...

Homologous Recombination

Homologous Recombination

The basic reaction of homologous recombination (HR) involves two chromatids that contain DNA sequences sharing a significant stretch of identity. One of these sequences uses a strand from another as a template to synthesize DNA in an enzyme-catalyzed reaction. The final product is a novel amalgamation of the two substrates. To ensure an accurate recombination of sequences, HR is restricted to the S and G2 phases of the cell cycle. At these stages, the DNA has been replicated already and the...

What is Genetic Engineering?

What is Genetic Engineering?

Conservative Site-specific Recombination and Phase Variation

Conservative Site-specific Recombination and Phase Variation

Because the DNA segments are cut and reorganized in a direction-specific manner, site-specific recombination has emerged as an efficient genetic engineering technique. Flippase and Cyclization recombinases or Flp and Cre, respectively, are two members of the tyrosine recombinase family derived from bacteriophages, that are used to mediate site-specific DNA insertions, deletions, and targeted expression of proteins in mammalian cell lines.
The recognition sites for Cre recombinase called LoxP...

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Related Experiment Video

Updated: Jun 18, 2025

A Standard Methodology to Examine On-site Mutagenicity As a Function of Point Mutation Repair Catalyzed by CRISPR/Cas9 and SsODN in Human Cells

A Standard Methodology to Examine On-site Mutagenicity As a Function of Point Mutation Repair Catalyzed by CRISPR/Cas9 and SsODN in Human Cells

Published on: August 25, 2017

CRISPR technology in human diseases.

Qiang Feng^1,2, Qirong Li¹, Hengzong Zhou¹

¹Laboratory Animal Center College of Animal Science Jilin University Changchun China.

|July 31, 2024

Summary

This summary is machine-generated.

Gene editing technology, particularly CRISPR-based systems, offers promising therapeutic strategies for various human diseases and aids in creating disease models. This approach enables precise genetic modifications for potential cures and improved treatments.

Keywords:

CRISPR–Cas9 clinical research gene editing technology gene therapy human diseases sickle cell disease

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Area of Science:

Biotechnology
Genetics
Molecular Biology

Background:

Gene editing is an evolving gene engineering technique with potential for curative treatments.
CRISPR-Cas systems enable precise genetic modifications in somatic cells, advancing gene therapy.
Gene editing shows promise in both treating diseases and developing animal models for human conditions.

Purpose of the Study:

To describe the applications of gene editing technology in various human diseases.
To focus on therapeutic strategies for sickle cell disease using gene editing.
To provide an overview of gene editing in constructing animal models and discuss its limitations.

Main Methods:

Review of current gene editing applications in hematological diseases, solid tumors, immune disorders, ophthalmological diseases, and metabolic diseases.
Focus on CRISPR-Cas systems for genetic modification in somatic cells.
Analysis of gene editing's role in disease modeling and therapeutic strategies.

Main Results:

Gene editing demonstrates significant potential across a spectrum of human diseases.
Specific therapeutic strategies are highlighted for sickle cell disease.
The technology is effective in creating animal models for human diseases.

Conclusions:

Gene editing technology, especially CRISPR, presents a powerful new strategy for human disease gene therapy.
Its applications extend to developing disease models and exploring novel therapeutic avenues.
Understanding the limitations is crucial for advancing gene editing in clinical practice.