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Related Concept Videos

CRISPR01:59

CRISPR

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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...
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CRISPR/Cas9 Genome Editing01:28

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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...
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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.
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Gene Therapy00:59

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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...
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CRISPR and crRNAs02:53

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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.
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Updated: Dec 31, 2025

Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms
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Sharpening the Molecular Scissors: Advances in Gene-Editing Technology.

Mike Broeders1, Pablo Herrero-Hernandez1, Martijn P T Ernst1

  • 1Department of Pediatrics, Erasmus University Medical Center, 3015 GD Rotterdam, Netherlands; Department of Clinical Genetics, Erasmus University Medical Center, 3015 GD Rotterdam, Netherlands; Center for Lysosomal and Metabolic Diseases, Erasmus University Medical Center, 3015 GE Rotterdam, Netherlands.

Iscience
|January 6, 2020
PubMed
Summary

Gene editing tools like CRISPR/Cas enable precise DNA modifications for various applications. Advances in accuracy, delivery, and clinical trials show great promise for treating genetic disorders and diseases.

Keywords:
GeneticsMolecular BiologyTechniques in Genetics

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Area of Science:

  • Molecular Biology
  • Genetics
  • Biotechnology

Background:

  • Gene editing technologies, including meganucleases, zinc finger nucleases, TALENs, and CRISPR/Cas, offer precise control over human gene modification.
  • These tools facilitate targeted genetic alterations such as deletions, insertions, gene knockouts, and point variants, as well as gene expression modulation and RNA targeting.

Purpose of the Study:

  • To review technological advancements in gene editing.
  • To discuss the endogenous DNA repair mechanisms utilized for gene modification.
  • To explore strategies for monitoring and minimizing off-target effects and alternative gene editing systems.

Main Methods:

  • Utilizes endogenous DNA repair pathways: non-homologous end joining, homology-directed repair, and others.
  • Employs in silico prediction and sequencing for off-target effect monitoring.
  • Investigates alternative Cas proteins (Cpf1, Cas12a, Cas12b, CjCas9) and high-fidelity Cas9 variants.
  • Examines ex vivo and in vivo delivery methods (AAV, lipid nanoparticles, cell-penetrating peptides).

Main Results:

  • Gene editing enables precise genetic modifications and gene expression modulation.
  • Various endogenous repair mechanisms are harnessed for DNA editing.
  • Strategies exist to enhance accuracy and monitor off-target effects.
  • Diverse delivery methods are available for gene editing components.

Conclusions:

  • Gene editing technology is advancing rapidly with improved accuracy and delivery systems.
  • Clinical applications are progressing for cancer immunotherapy, HIV treatment, and genetic disorders like beta-thalassemia and sickle cell disease.
  • Challenges remain in clinical implementation, necessitating continued research and development.