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Related Concept Videos

CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

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...
CRISPR01:59

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 Short...
CRISPR01:59

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 Short...
Homologous Recombination02:31

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...
CRISPR and crRNAs02:53

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...
Overview of DNA Repair02:25

Overview of DNA Repair

In order to be passed through generations, genomic DNA must be undamaged and error-free. However, every day, DNA in a cell undergoes several thousand to a million damaging events by natural causes and external factors. Ionizing radiation such as UV rays, free radicals produced during cellular respiration, and hydrolytic damage from metabolic reactions can alter the structure of DNA. Damages caused include single-base alteration, base dimerization, chain breaks, and cross-linkage.
Chemically...

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Related Experiment Video

Updated: Jul 16, 2026

A Standard Methodology to Examine On-site Mutagenicity As a Function of Point Mutation Repair Catalyzed by CRISPR/Cas9 and SsODN in Human Cells
10:07

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/Cas9-Based Genome Editing: Understanding Differences in DNA Repair Pathways, Profiles, and Outcomes.

Samuel N Effah1,2, Shirley C Barrera1,2, Nahia Urturi Ortiz1,2

  • 1Department of Microbiology and Immunology, Drexel University College of Medicine, Philadelphia, PA 19102, USA.

International Journal of Molecular Sciences
|July 15, 2026
PubMed
Summary

CRISPR/Cas9 gene editing, now FDA-approved for sickle cell anemia, shows promise for chronic diseases like HIV-1. Success depends on delivery, editing efficiency, and repair outcomes, with new analytical tools aiding prediction.

Keywords:
C-NHEJCRISPR/Cas9HIV-1MMEJchromatinindelprediction and analytical toolsrepair editrepair outcomerepair profiletarget sequence

More Related Videos

Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms
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Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms

Published on: May 25, 2018

CIRCLE-Seq for Interrogation of Off-Target Gene Editing
08:23

CIRCLE-Seq for Interrogation of Off-Target Gene Editing

Published on: November 1, 2024

Related Experiment Videos

Last Updated: Jul 16, 2026

A Standard Methodology to Examine On-site Mutagenicity As a Function of Point Mutation Repair Catalyzed by CRISPR/Cas9 and SsODN in Human Cells
10:07

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

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

CIRCLE-Seq for Interrogation of Off-Target Gene Editing
08:23

CIRCLE-Seq for Interrogation of Off-Target Gene Editing

Published on: November 1, 2024

Area of Science:

  • Biotechnology
  • Gene Editing
  • Molecular Biology

Background:

  • Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR) and CRISPR-associated protein 9 (Cas9) technologies have advanced significantly over the past decade.
  • This progress led to the first FDA-approved CRISPR/Cas-based therapy for sickle cell anemia.
  • Numerous CRISPR/Cas-based therapies are in development for chronic conditions including HIV-1, familial hypercholesterolemia, and cancer.

Purpose of the Study:

  • To discuss factors influencing CRISPR/Cas9-generated repair edits and their outcomes.
  • To review analytical tools developed for assessing CRISPR/Cas9 editing.
  • To explore the application of CRISPR/Cas9 technology for a functional HIV-1 cure.

Main Methods:

  • Review of factors affecting CRISPR/Cas9 editing efficiency and repair profiles.
  • Discussion of analytical tools for evaluating gene editing outcomes.
  • Examination of CRISPR/Cas9 applications in treating chronic diseases, particularly HIV-1.

Main Results:

  • CRISPR/Cas9 therapy is now FDA-approved for sickle cell anemia.
  • Ongoing development of CRISPR/Cas9 therapies for viral, genetic, and cancerous diseases.
  • Effective delivery, editing, and repair are crucial for therapeutic success.

Conclusions:

  • CRISPR/Cas9 technology holds significant therapeutic potential across various chronic diseases.
  • Understanding and predicting repair outcomes are key to successful gene editing therapies.
  • CRISPR/Cas9 offers a promising avenue towards a functional cure for HIV-1.