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Related Concept Videos

CRISPR01:59

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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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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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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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Selection-dependent and Independent Generation of CRISPR/Cas9-mediated Gene Knockouts in Mammalian Cells
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In vivo genome editing using Staphylococcus aureus Cas9.

F Ann Ran1, Le Cong2, Winston X Yan3

  • 11] Broad Institute of MIT and Harvard, Cambridge, Massachusetts 02142, USA [2] Society of Fellows, Harvard University, Cambridge, Massachusetts 02138, USA.

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Summary

Smaller Staphylococcus aureus Cas9 (SaCas9) enables efficient genome editing via adeno-associated virus vectors. This SaCas9 system successfully modified the Pcsk9 gene in mice, reducing cholesterol levels with high specificity.

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Area of Science:

  • Molecular Biology
  • Biotechnology
  • Genetics

Background:

  • The Cas9 enzyme is a powerful tool for genome editing.
  • The large size of Streptococcus pyogenes Cas9 (SpCas9) restricts its use with adeno-associated virus (AAV) delivery systems.
  • Smaller Cas9 variants are needed for efficient AAV-mediated gene editing.

Purpose of the Study:

  • To characterize smaller Cas9 orthologues for genome editing.
  • To evaluate the efficiency and specificity of Staphylococcus aureus Cas9 (SaCas9) for in vivo gene editing.
  • To demonstrate the therapeutic potential of SaCas9 using AAV delivery.

Main Methods:

  • Characterization of six smaller Cas9 orthologues.
  • In vivo genome editing in mouse liver targeting the Pcsk9 gene using SaCas9 delivered via AAV.
  • Assessment of gene modification efficiency and reduction in serum Pcsk9 and cholesterol levels.
  • Genome-wide specificity analysis using BLESS (Binding and Ligation-based Exonuclease Sequencing).

Main Results:

  • Staphylococcus aureus Cas9 (SaCas9) demonstrated genome editing efficiencies comparable to SpCas9.
  • SaCas9 is over 1 kilobase shorter than SpCas9, facilitating AAV packaging.
  • In vivo SaCas9 delivery resulted in >40% Pcsk9 gene modification in mouse liver within one week.
  • Significant reductions in serum Pcsk9 and total cholesterol levels were observed post-editing.
  • SaCas9 exhibited efficient and specific genome-wide targeting in vivo.

Conclusions:

  • SaCas9 is a viable, smaller alternative to SpCas9 for genome editing applications.
  • AAV-packaged SaCas9 enables efficient in vivo gene editing with therapeutic potential.
  • SaCas9-mediated editing offers a promising strategy for managing conditions like hypercholesterolemia.