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Related Concept Videos

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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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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The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
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Transducer Mechanism: Enzyme-Linked Receptors01:27

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Enzyme-linked receptors are cell-surface receptors acting as an enzyme or associating with an enzyme intracellularly. They make excellent drug targets. Drugs can bind to the extracellular ligand-binding domain or directly affect their enzymatic domain and alter their activity.
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The ability of induced pluripotent stem cells or iPSCs to differentiate into most body cell types has stimulated repair and regenerative medicine research over the past few decades. iPSC-derived blood cells, hepatocytes, beta islet cells, cardiomyocytes, neurons, and other cell types can repair injuries or regenerate damaged tissue in diseases such as diabetes and neurodegenerative disorders.
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Related Experiment Video

Updated: Oct 6, 2025

A High-Throughput Luciferase Assay to Evaluate Proteolysis of the Single-Turnover Protease PCSK9
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Gene Therapy Targeting PCSK9.

Julius L Katzmann1, Arjen J Cupido2,3,4, Ulrich Laufs1

  • 1Department of Cardiology, University Hospital Leipzig, 04103 Leipzig, Germany.

Metabolites
|January 20, 2022
PubMed
Summary
This summary is machine-generated.

Gene editing using CRISPR/Cas offers a potential one-time treatment for lowering LDL cholesterol, aiming for long-term cardiovascular prevention without side effects or regular medication.

Keywords:
CRISPR/CasLDL cholesterolPCSK9base editingcardiovascular diseasegene editinghypercholesterolemiain vivo

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Area of Science:

  • Cardiovascular Science
  • Genetics
  • Molecular Biology

Background:

  • Cardiovascular prevention research has advanced LDL hypercholesterolemia treatments.
  • PCSK9 inhibitors (monoclonal antibodies, RNA drugs) are recent developments.
  • Durable LDL cholesterol reduction is desired for atherosclerosis prevention.

Purpose of the Study:

  • To review the application of CRISPR/Cas gene editing for cardiovascular prevention.
  • To present the current state of in vivo PCSK9 editing.

Main Methods:

  • CRISPR/Cas system for precise genome editing.
  • In vivo gene or base editing approaches targeting PCSK9.
  • Review of current research on PCSK9 editing in primates.

Main Results:

  • Durable LDL cholesterol reductions observed in primates after single PCSK9 gene/base editor treatment.
  • CRISPR/Cas enables precise genome editing, including single-nucleotide changes.

Conclusions:

  • PCSK9 gene editing shows potential for durable, long-term LDL cholesterol reduction.
  • CRISPR/Cas technology could fundamentally alter cardiovascular prevention strategies.
  • Safety and cardiovascular event reduction documentation are crucial for clinical translation.