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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

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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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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What is Genetic Engineering?00:49

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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: Aug 14, 2025

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CRISPR/Cas9 therapeutics: progress and prospects.

Tianxiang Li1, Yanyan Yang2, Hongzhao Qi1

  • 1Institute for Translational Medicine, The Affiliated Hospital of Qingdao University, No. 38 Dengzhou Road, 266021, Qingdao, People's Republic of China.

Signal Transduction and Targeted Therapy
|January 16, 2023
PubMed
Summary
This summary is machine-generated.

Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) gene editing offers precise disease treatment. Effective in vivo delivery of CRISPR/Cas9 systems remains a key challenge for future therapeutic development.

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Area of Science:

  • Biotechnology
  • Genetics
  • Molecular Biology

Background:

  • CRISPR/Cas9 gene editing technology shows great promise for permanent disease correction and gene disruption.
  • Development of efficient Cas9 variants addresses complex genomic alterations in diseases.
  • Current limitations exist in the effective in vivo delivery of CRISPR systems to target cells.

Purpose of the Study:

  • To review current developments in CRISPR/Cas9 gene editing for disease treatment.
  • To examine gene-editing types, delivery vectors, and disease characteristics relevant to CRISPR applications.
  • To summarize clinical trial successes and identify challenges in CRISPR/Cas9 targeted delivery.

Main Methods:

  • Review of CRISPR/Cas9 gene-editing technologies and their variants.
  • Analysis of nonviral delivery vectors with target recognition capabilities.
  • Examination of disease-specific factors for targeted delivery and gene editing strategies.

Main Results:

  • CRISPR/Cas9 technology enables precise correction of mutations and disruption of disease genes.
  • Nonviral vectors with targeting functions are a focus for future in vivo delivery research.
  • Disease characteristics can inform targeted delivery and gene editing approaches.

Conclusions:

  • Effective in vivo delivery remains a significant hurdle for CRISPR/Cas9-based disease therapies.
  • Tailoring gene-editing methods and delivery vectors to specific diseases is crucial.
  • Further research is needed to overcome challenges in targeted delivery and clinical application of CRISPR/Cas9.