Related Experiment Video
Updated: Jan 29, 2026
07:56
Using CRISPR/Cas9 to Knock Out GM-CSF in CAR-T Cells
Published on: July 22, 2019
11.9K
CRISPR/Cas9 for Sickle Cell Disease: Applications, Future Possibilities, and Challenges
Selami Demirci1,2, Alexis Leonard3, Juan J Haro-Mora3
1Cellular and Molecular Therapeutics Branch, NHLBI/NIDDK, National Institutes of Health, Bethesda, MD, USA. selami.demirci@nih.gov.
Advances in Experimental Medicine and Biology
|February 5, 2019
Summary
Genome editing with CRISPR/Cas9 offers a promising curative strategy for sickle cell disease (SCD). This technology can correct the genetic mutation or induce fetal hemoglobin to combat red blood cell sickling.
Area of Science:
- Genetics
- Molecular Biology
- Hematology
Background:
- Sickle cell disease (SCD) is a debilitating inherited blood disorder with limited treatment options.
- Despite over a century of research, a universal cure for SCD remains elusive, with only two FDA-approved drugs available.
Purpose of the Study:
- To summarize the application of CRISPR/Cas9 genome editing for sickle cell disease.
- To discuss the challenges and future outlook of CRISPR/Cas9 as a potential cure for SCD.
Main Methods:
- Review of CRISPR/Cas9 genome engineering strategies for SCD.
- Focus on correcting the causative mutation in hematopoietic stem/progenitor cells (HSPCs).
- Exploration of inducing fetal hemoglobin (HbF) expression to mitigate red blood cell sickling.
Main Results:
- CRISPR/Cas9 has revolutionized genome engineering, offering potential therapeutic avenues for SCD.
- Genome editing can correct the underlying mutation or compensate for it by increasing HbF levels.
- Development of corrected induced pluripotent stem cells (iPSCs) is another viable approach.
Conclusions:
- CRISPR/Cas9-based genome editing presents a significant advancement towards a definitive cure for sickle cell disease.
- Further research and clinical translation are necessary to overcome challenges and realize the full potential of this technology for SCD patients.
Related Concept Videos
01:59CRISPR
57.8K
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...
57.8K
02:53CRISPR and crRNAs
19.1K
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...
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...
19.1K
01:28CRISPR/Cas9 Genome Editing
1.8K
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...
1.8K
01:21EPS and iPS Cells in Disease Research
3.4K
Embryonic and induced pluripotent stem cells are excellent models for disease research because of their ability to self-renew and differentiate into most cell types. Somatic cells from a patient are isolated and reprogrammed into induced pluripotent stem cells or iPSCs. These iPSCs are later differentiated into the desired cell type, which mirrors the diseased cell of the patient. In this way, disease models have been created for investigating diseases such as Down syndrome, type I diabetes,...
3.4K
01:19Clinical Applications of Epidermal Stem Cells
3.3K
Epidermal stem cells (EpiSCs) are mainly located at the basal layer of the epidermis. These cells repair minor injuries of the skin and replace dead skin cells. However, EpiSCs’ cannot heal severe wounds such as major burns or those from diabetes or hereditary disorders. In such cases, culturing the epidermal stem cells from the patient is possible and has yielded successful treatment options, such as laboratory-grown skin grafts. These grafts are synthesized using a patient’s own...
3.3K
01:23The Antiviral System of Bacteria and Archaea: CRISPR
689
CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats is a adaptive immune system found in bacteria and archaea that protects against viral infections. This system enables prokaryotic cells to identify, remember, and neutralize foreign genetic elements, primarily bacteriophages, by storing fragments of the invader’s DNA as a genetic memory.The CRISPR immune response begins during an initial infection. Cas (CRISPR-associated) proteins play a central role in this...
689