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Related Concept Videos

CRISPR01:59

CRISPR

58.4K
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...
58.4K
CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

2.2K
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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Related Experiment Video

Updated: Mar 5, 2026

Genome Editing in Mammalian Cell Lines using CRISPR-Cas
07:56

Genome Editing in Mammalian Cell Lines using CRISPR-Cas

Published on: April 11, 2019

23.4K

Delivery technologies for genome editing.

Hao Yin1, Kevin J Kauffman1,2, Daniel G Anderson1,2,3,4

  • 1David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, Massachusetts 02139, USA.

Nature Reviews. Drug Discovery
|March 25, 2017
PubMed
Summary
This summary is machine-generated.

Genome editing tools like CRISPR are advancing rapidly. Efficient and safe delivery methods are crucial for their use in research and gene therapy, with ongoing clinical trials exploring these applications.

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Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms

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Related Experiment Videos

Last Updated: Mar 5, 2026

Genome Editing in Mammalian Cell Lines using CRISPR-Cas
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Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms
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Area of Science:

  • Biotechnology
  • Molecular Biology
  • Genomics

Background:

  • CRISPR, TALENs, and ZFNs are powerful genome engineering tools.
  • These technologies have applications in biomedical research, drug discovery, and gene therapy.
  • Effective cellular delivery is essential for the function of genome editing systems.

Purpose of the Study:

  • To review the principles of gene editing and biomacromolecule delivery.
  • To examine recent advancements and challenges in viral and non-viral delivery methods.
  • To highlight the current status of clinical trials involving gene editing technologies.

Main Methods:

  • Review of scientific literature on genome editing and delivery systems.
  • Analysis of current non-viral and viral delivery strategies.
  • Compilation of data on ongoing clinical trials.

Main Results:

  • Genome editing technologies are increasingly accessible.
  • Delivery methods remain a critical challenge for clinical translation.
  • Various delivery strategies are under investigation, each with unique advantages and limitations.

Conclusions:

  • Efficient and safe delivery is key to realizing the full potential of genome editing.
  • Continued research in delivery technologies is vital for advancing gene therapy and biomedical applications.
  • Clinical trials are progressing, indicating the therapeutic potential of these engineered nucleases.