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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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What is Genetic Engineering?00:49

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Related Experiment Video

Updated: Jun 21, 2025

Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice
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Gene editing therapy for cardiovascular diseases.

Xinyu Wu1, Jie Yang1, Jiayao Zhang1

  • 1State Key Laboratory for Diagnosis and Treatment of Severe Zoonotic Infectious Diseases Key Laboratory for Zoonosis Research of the Ministry of Education and College of Veterinary Medicine Jilin University Changchun China.

Medcomm
|July 8, 2024
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Summary

Gene editing tools, including CRISPR/Cas, offer promising new therapies for cardiovascular disease (CVD). Research is advancing rapidly, showing potential for clinical applications and improved patient outcomes in CVD treatment.

Keywords:
CRISPR/Cascardiovascular diseasegene therapylipid nanoparticles

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Area of Science:

  • Life Sciences
  • Biotechnology
  • Genetics

Background:

  • Gene editing tools have evolved significantly over 30 years.
  • These technologies are crucial for disease detection and research.
  • Recent advancements highlight their therapeutic potential, especially for cardiovascular disease (CVD).

Purpose of the Study:

  • To provide an overview of DNA-targeted gene editing tools.
  • To focus on the application of gene editing, particularly CRISPR/Cas, in CVD therapy.
  • To summarize challenges, limitations, and ongoing research in CVD gene editing.

Main Methods:

  • Review of established DNA-targeted gene editing technologies.
  • Specific focus on the CRISPR/Cas system's role in cardiovascular research.
  • Analysis of current clinical trials and research progress in CVD gene editing.

Main Results:

  • Gene editing therapies are demonstrating significant potential for treating CVD.
  • The CRISPR/Cas system is a key technology being explored for CVD applications.
  • Progress reflects the integration of gene editing in both basic research and clinical settings.

Conclusions:

  • Gene editing therapies hold considerable promise for the future of cardiovascular disease treatment.
  • Continued research and clinical trials are essential to overcome current challenges.
  • Successful applications of gene editing in CVD pave the way for further innovation.