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Related Concept Videos

Gene Therapy00:59

Gene Therapy

27.9K
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...
27.9K

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Related Experiment Video

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Gene Transfer for Ischemic Heart Failure in a Preclinical Model
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Current Methods in Cardiac Gene Therapy: Overview.

Kiyotake Ishikawa1, Roger J Hajjar2

  • 1Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, One Gustave L. Levy Place, Box 1030, New York, NY, 10029-6574, USA.

Methods in Molecular Biology (Clifton, N.J.)
|December 3, 2016
PubMed
Summary
This summary is machine-generated.

Cardiac gene therapy shows promise but faces challenges in humans. Refined delivery tools and methods are crucial for future clinical success in treating heart conditions.

Keywords:
Adeno-associated vectorsCardiac gene therapyCardiotropicGene deliveryHeart failurePercutaneous deliveryPromotersSurgical delivery

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Area of Science:

  • Cardiovascular Medicine
  • Molecular Biology
  • Biotechnology

Background:

  • Cardiac gene therapy has advanced significantly from preclinical data, with early trials showing promise.
  • Recent larger trials yielded neutral results, highlighting challenges in clinical translation.
  • Despite setbacks, valuable knowledge has been gained, driving innovation in vectors and delivery.

Purpose of the Study:

  • To provide an overview of the current status of cardiac gene therapy.
  • To focus on the critical aspects of gene delivery tools and methods.
  • To discuss advancements expected to improve future clinical outcomes.

Main Methods:

  • Review of preclinical and clinical trial data in cardiac gene therapy.
  • Analysis of gene delivery vectors and their efficacy.
  • Examination of technological and methodological advancements.

Main Results:

  • Early phase trials demonstrated encouraging results, but larger trials reported neutral outcomes.
  • Humans exhibit greater resistance to therapeutic transgene expression than animals.
  • Progress is being made in developing cardiotropic vectors and improved delivery systems.

Conclusions:

  • Cardiac gene therapy requires refined gene delivery tools and methods for success in humans.
  • Newer technologies and approaches are anticipated to yield more promising clinical results.
  • Continued research and development are essential for advancing cardiac gene therapy.