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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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Stem Cell Therapy for Tissue Regeneration01:21

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Stem cell therapy is a method used in regenerative medicine to repair and restore function to damaged tissues and organs. Stem cells have the potential to proliferate and differentiate into various tissue types, making them ideal candidates for tissue regeneration. For example, hematopoietic stem cell transplants are commonly used in blood cancer treatment to replenish damaged bone marrow and restore healthy blood cells.
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Microorganisms in Medicine and Therapeutics01:29

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Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.
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iPS Cell Differentiation01:22

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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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Stem cell research aims to find ways to use stem cells to regenerate and repair cellular damage. Over time, most adult cells undergo the wear and tear of aging and lose their ability to divide and repair themselves. Stem cells do not display a particular morphology or function. Adult stem cells, which exist as a small subset of cells in most tissues, keep dividing and can differentiate into a number of specialized cells generally formed by that tissue. These cells enable the body to renew and...
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Gene Therapy for Regenerative Medicine.

Hossein Hosseinkhani1, Abraham J Domb2, Ghorbanali Sharifzadeh3

  • 1Innovation Center for Advanced Technology, Matrix, Inc., New York, NY 10019, USA.

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|March 29, 2023
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Summary

Gene therapy and tissue engineering advance human tissue regeneration. This review focuses on non-viral gene delivery methods and tissue engineering for improved regenerative medicine therapies.

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biodegradable polymersgene therapynanoparticlesnon-viral vectorsregenerative medicinetissue engineeringviral vectors

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Area of Science:

  • Regenerative Medicine
  • Gene Therapy
  • Tissue Engineering

Background:

  • Recent decades show significant progress in biological methods for human tissue regeneration.
  • Stem cell research, gene therapy, and tissue engineering are key drivers of this advancement.
  • Clinical application of gene therapy faces technical challenges, particularly regarding safety and efficacy.

Purpose of the Study:

  • To critically review gene therapy approaches for regenerative medicine.
  • To highlight the potential of non-viral gene transfection agents as safer alternatives to viral vectors.
  • To explore the integration of tissue engineering technologies for enhanced in vivo gene delivery and function.

Main Methods:

  • Review of current literature on gene therapy and regenerative medicine.
  • Analysis of viral and non-viral gene delivery systems.
  • Evaluation of tissue engineering strategies for therapeutic gene delivery.

Main Results:

  • Non-viral gene transfection agents show promise for treating genetic and acquired diseases, offering potential safety advantages over viral vectors.
  • Enhancing the efficiency of non-viral vectors to match viral vector performance is a key research focus.
  • Tissue engineering offers a promising avenue to improve the control over the in vivo location and function of administered genes.

Conclusions:

  • Integrating tissue engineering with gene therapy can overcome limitations of current viral and non-viral approaches.
  • Further development of non-viral vectors and tissue engineering strategies is crucial for advancing regenerative medicine.
  • Controlling gene delivery and function in vivo is essential for successful therapeutic outcomes in regenerative medicine.