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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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What is Genetic Engineering?00:49

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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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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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Precision modification of heart failure signaling by CRISPR-Cas9 base editing.

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Related Experiment Video

Updated: Sep 15, 2025

Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice
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Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice

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Gene Editing Applications as Future Cardiovascular Therapies.

Tomonori Tadokoro1,2, Eric N Olson1,2, Ning Liu1,2

  • 1Department of Molecular Biology, University of Texas Southwestern Medical Center, Dallas, Texas, USA; email: Tomonori.Tadokoro@UTSouthwestern.edu, Eric.Olson@UTSouthwestern.edu, Ning.Liu@UTSouthwestern.edu.

Annual Review of Genetics
|July 16, 2025
PubMed
Summary
This summary is machine-generated.

CRISPR-Cas9 gene editing shows promise for treating cardiovascular diseases by correcting genetic defects. Further research is needed to overcome delivery and safety challenges for clinical use.

Keywords:
CRISPR-Cas9RNA editingadeno-associated viruscardiomyopathygene editingnanoparticle

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Area of Science:

  • Biotechnology
  • Genetics
  • Cardiology

Background:

  • Cardiovascular disease is a major global health burden.
  • Current treatments have limitations.
  • CRISPR-Cas9 offers a novel therapeutic avenue.

Purpose of the Study:

  • To review gene editing strategies for cardiovascular disorders.
  • To discuss therapeutic applications, merits, and limitations.
  • To explore regulatory considerations for clinical translation.

Main Methods:

  • Review of current CRISPR-Cas9 gene editing technologies.
  • Analysis of potential applications in cardiovascular disease.
  • Evaluation of delivery methods and safety profiles.

Main Results:

  • CRISPR-Cas9 can correct genetic defects and modulate disease pathways.
  • In vivo delivery and safety are key challenges.
  • Significant therapeutic potential exists for various heart conditions.

Conclusions:

  • Gene editing holds transformative potential for cardiovascular medicine.
  • Addressing technical and safety hurdles is crucial for clinical adoption.
  • Continued advancements promise future therapeutic breakthroughs.