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Related Concept Videos

CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

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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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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 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.
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...
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Homologous Recombination02:31

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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...
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Updated: Mar 19, 2026

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

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Optimization of genome editing through CRISPR-Cas9 engineering.

Jian-Hua Zhang1, Poorni Adikaram1, Mritunjay Pandey1

  • 1a Metabolic Diseases Branch , National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health , Bethesda , MD , USA.

Bioengineered
|June 25, 2016
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CRISPR-Cas9 gene editing offers revolutionary medical potential but faces challenges like off-target effects and variable efficiency. Further research is crucial to perfect this powerful genome editing tool for clinical use.

Keywords:
CRISPR-Cas9efficiencygenome editingspecificity

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Area of Science:

  • Molecular Biology
  • Genetics
  • Biotechnology

Background:

  • CRISPR-Cas9 is a powerful genome editing tool with significant medical potential.
  • It utilizes a guide RNA (gRNA) to target and cut specific DNA sequences.
  • DNA repair mechanisms like NHEJ and HDR enable gene modification.

Purpose of the Study:

  • To review the current state of CRISPR-Cas9 technology.
  • To identify existing challenges and recent advancements.
  • To offer perspectives on improving CRISPR-Cas9 for medical applications.

Main Methods:

  • Review of CRISPR-Cas9 mechanisms, including DNA targeting and repair pathways.
  • Analysis of off-target effects, efficiency variations, and PAM limitations.
  • Synthesis of current research addressing these challenges.

Main Results:

  • CRISPR-Cas9 enables precise genome editing but suffers from off-target mutations.
  • Efficiency varies across different DNA targets, and PAM recognition restricts its use.
  • Ongoing research aims to enhance specificity, efficiency, and broaden targeting capabilities.

Conclusions:

  • CRISPR-Cas9 technology holds immense promise for medicine.
  • Overcoming off-target activity and efficiency issues is critical for clinical translation.
  • Continued innovation is necessary to fully realize the potential of Cas9-mediated genome editing.