Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

Related Concept Videos

CRISPR01:59

CRISPR

55.0K
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...
55.0K
CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

1.0K
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...
1.0K
Homologous Recombination02:31

Homologous Recombination

59.2K
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...
59.2K
CRISPR and crRNAs02:53

CRISPR and crRNAs

18.2K
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...
18.2K
What is Genetic Engineering?00:49

What is Genetic Engineering?

77.3K
Overview
77.3K

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by
Same author

Comparison Between Titanium and Polyether Ether Ketone Cranioplasty Based on Preoperative Imaging Indicators and Analysis of Risk Factors for Postoperative Adverse Events.

World neurosurgery·2026
Same author

Systematic analyses to explore kinase gene set-based signature in glioma, in which WEE1 contributes to tumor progression.

Discover oncology·2026
Same author

Sequential manifestation of Kaposi's sarcoma and diffuse large B-cell lymphoma in the context of HIV infection: Case report of a rare presentation.

Medicine·2026
Same author

Focal Adhesion Related Gene Signature for Glioma: The Pivotal Role of RAP1B in Disease Progression.

Current medicinal chemistry·2026
Same author

High-power dual-channel chamber for high-frequency magnetic neuromodulation.

Journal of neural engineering·2026
Same author

Cytosine base editing of LPA in transgenic mice averts large deletions.

Molecular therapy : the journal of the American Society of Gene Therapy·2026

Related Experiment Video

Updated: Nov 21, 2025

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
08:32

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications

Published on: August 9, 2022

4.1K

CRISPR/Cas9 gene editing for curing sickle cell disease.

So Hyun Park1, Gang Bao1

  • 1Department of Bioengineering, Rice University, 6500 Main St, Houston, TX, 77030, USA.

Transfusion and Apheresis Science : Official Journal of the World Apheresis Association : Official Journal of the European Society for Haemapheresis
|January 18, 2021
PubMed
Summary

Gene editing offers a potential cure for sickle cell disease (SCD) by correcting the HBB gene mutation or inducing fetal hemoglobin. This approach could provide a permanent solution for all SCD patients.

Keywords:
CRISPR/Cas9Gene editingSickle cell disease

More Related Videos

Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms
09:51

Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms

Published on: May 25, 2018

34.9K
Using CRISPR/Cas9 Gene Editing to Investigate the Oncogenic Activity of Mutant Calreticulin in Cytokine Dependent Hematopoietic Cells
10:21

Using CRISPR/Cas9 Gene Editing to Investigate the Oncogenic Activity of Mutant Calreticulin in Cytokine Dependent Hematopoietic Cells

Published on: January 5, 2018

13.5K

Related Experiment Videos

Last Updated: Nov 21, 2025

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
08:32

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications

Published on: August 9, 2022

4.1K
Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms
09:51

Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms

Published on: May 25, 2018

34.9K
Using CRISPR/Cas9 Gene Editing to Investigate the Oncogenic Activity of Mutant Calreticulin in Cytokine Dependent Hematopoietic Cells
10:21

Using CRISPR/Cas9 Gene Editing to Investigate the Oncogenic Activity of Mutant Calreticulin in Cytokine Dependent Hematopoietic Cells

Published on: January 5, 2018

13.5K

Area of Science:

  • Hematology
  • Genetics
  • Molecular Biology

Background:

  • Sickle cell disease (SCD) is a prevalent monogenic blood disorder causing severe pain, organ damage, and reduced lifespan.
  • Current treatments for SCD are limited, with hematopoietic stem cell transplantation being the only cure, often requiring a matched donor.

Purpose of the Study:

  • To review the application of CRISPR/Cas9 gene-editing technology for curing sickle cell disease.
  • To explore two primary gene-editing strategies: direct correction of the HBB mutation and induction of fetal hemoglobin.

Main Methods:

  • Focuses on CRISPR/Cas9 gene-editing techniques applied to hematopoietic stem and progenitor cells.
  • Discusses ex vivo genetic modification of autologous cells for subsequent transplantation.

Main Results:

  • CRISPR/Cas9 shows promise in correcting the SCD-causing mutation in the beta-globin (HBB) gene.
  • Gene editing can induce fetal hemoglobin production, effectively reversing red blood cell sickling.

Conclusions:

  • CRISPR/Cas9 gene editing presents a potential curative therapy for SCD, overcoming donor limitations and graft-versus-host disease.
  • Further research and development are crucial to address challenges and realize the full potential of gene-editing therapies for SCD.