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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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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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To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.
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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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Gene Therapy00:59

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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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Related Experiment Video

Updated: Jun 18, 2025

Electroporation-Mediated Delivery of Cas9 Ribonucleoproteins and mRNA into Freshly Isolated Primary Mouse Hepatocytes
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Gene editing in liver diseases.

Laura Torella1, Nerea Santana-Gonzalez1, Nerea Zabaleta2

  • 1DNA & RNA Medicine Division, Gene Therapy for Rare Diseases Department, Center for Applied Medical Research (CIMA), University of Navarra, IdisNA, Pamplona, Spain.

FEBS Letters
|July 30, 2024
PubMed
Summary
This summary is machine-generated.

Gene editing technologies like CRISPR Cas9 offer new ways to treat inherited liver diseases by modifying the genome. Early clinical results show promise, but more research is needed on efficacy and limitations.

Keywords:
gene editing clinical studiesgene editing preclinical studiesgene editing toolsinherited liver diseasesliver gene delivery vehicles

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Area of Science:

  • Genetics and genomics
  • Molecular biology
  • Medical biotechnology

Background:

  • Engineered nucleases enable precise host genome modification, advancing modern medicine.
  • CRISPR Cas9 technology has revolutionized in vivo gene editing for therapeutic applications.
  • Clinical trials for metabolic liver disorders show promising outcomes, notably in transthyretin amyloidosis.

Purpose of the Study:

  • To review preclinical data on gene editing in animal models of inherited liver diseases.
  • To summarize current clinical data on gene editing therapies for liver conditions.
  • To emphasize the therapeutic efficacy and potential limitations of these interventions.

Main Methods:

  • Review of preclinical studies involving gene editing in animal models.
  • Analysis of clinical trial data for gene editing therapies.
  • Synthesis of information on gene supplementation, correction, and silencing strategies.

Main Results:

  • Gene editing shows potential for treating metabolic liver disorders.
  • Transthyretin amyloidosis patients have shown remarkable outcomes with these therapies.
  • In vivo genome modification offers diverse therapeutic possibilities.

Conclusions:

  • Gene editing holds significant therapeutic potential for inherited liver diseases.
  • Further research is essential to fully understand the efficacy and limitations of gene editing interventions.
  • The field is rapidly evolving, with anticipated advancements in the coming years.