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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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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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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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RNA Editing02:23

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RNA editing is a post-transcriptional modification where a precursor mRNA (pre-mRNA) nucleotide sequence is changed by base insertion, deletion, or modification. The extent of RNA editing varies from a few hundred bases, in mitochondrial DNA of trypanosomes, to a just single base, in nuclear genes of mammals. Even a single base change in the pre-mRNA can convert a codon for one amino acid into the codon for another amino acid or a stop codon. This type of re-coding can significantly affect the...
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Gene Therapy00:59

Gene Therapy

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Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
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Cell Specific Gene Expression01:58

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Multicellular organisms contain a variety of structurally and functionally distinct cell types, but the DNA in all the cells originated from the same parent cells. The differences in the cells can be attributed to the differential gene expression. Liver cells, whose functions include detoxification of blood, production of bile to metabolize fats, and synthesis of proteins essential for metabolism, must express a specific set of genes to perform their functions. Gene expression also varies with...
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Related Experiment Video

Updated: Feb 12, 2026

CRISPR/Cas9 Ribonucleoprotein-mediated Precise Gene Editing by Tube Electroporation
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Efficient In Vivo Liver-Directed Gene Editing Using CRISPR/Cas9.

Kshitiz Singh1, Hanneke Evens1, Nisha Nair1

  • 1Department of Gene Therapy and Regenerative Medicine, Faculty of Medicine and Pharmacy, Vrije Universiteit Brussel (VUB), 1090 Brussels, Belgium.

Molecular Therapy : the Journal of the American Society of Gene Therapy
|March 31, 2018
PubMed
Summary

This study demonstrates successful liver-specific genome editing using CRISPR-Cas9 delivered via AAV9. The approach achieved efficient targeting of the Factor IX gene in mice, leading to a hemophilia B model.

Keywords:
AAVCRISPRCas9factor IXgRNAhepatocytesliveroff-targettruncated gRNA

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QTL Mapping and CRISPR/Cas9 Editing to Identify a Drug Resistance Gene in Toxoplasma gondii
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Highly Efficient Gene Disruption of Murine and Human Hematopoietic Progenitor Cells by CRISPR/Cas9
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Highly Efficient Gene Disruption of Murine and Human Hematopoietic Progenitor Cells by CRISPR/Cas9
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Area of Science:

  • Molecular Biology
  • Gene Editing
  • Hepatology

Background:

  • In vivo tissue-specific genome editing remains a significant challenge.
  • Targeting specific genes in the liver requires precise delivery and expression control.

Purpose of the Study:

  • To develop and evaluate a liver-specific CRISPR-Cas9 genome editing system for in vivo targeting.
  • To assess the efficiency and specificity of targeting the mouse Factor IX (F9) gene.

Main Methods:

  • Adeno-associated virus serotype 9 (AAV9) delivery of Cas9 and truncated guide RNAs (gRNAs).
  • Utilized a computationally designed hepatocyte-specific promoter for Cas9 expression control.
  • Assessed editing efficiency using T7E1 assays, Sanger sequencing, and deep sequencing.

Main Results:

  • Achieved robust liver-specific and sequence-specific targeting of the F9 gene.
  • Demonstrated high indel frequency (up to 50%) in the liver with no off-target effects observed in other tissues.
  • Observed a loss of Factor IX activity and a bleeding phenotype consistent with hemophilia B.

Conclusions:

  • AAV9-mediated delivery with a hepatocyte-specific promoter enables efficient and specific in vivo liver genome editing.
  • Transient Cas9 expression offers a "hit-and-run" gene editing paradigm with broad implications for somatic gene therapy in the liver.