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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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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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Nuclear reprogramming is the process of switching gene expression of one cell type to that of another cell type, usually from a differentiated cell state to an undifferentiated cell state. Differentiation occurs during processes such as development and morphogenesis, tissue regeneration, and malignancy. Cells can also be artificially induced to reprogram their gene expression by techniques such as nuclear transfer, induced pluripotency, and cell fusion. Such techniques have many applications in...
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Embryonic Stem Cells00:57

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Embryonic stem (ES) cells were first discovered in mice in 1981 by Martin Evans. In 1998, James Thomson identified a method to isolate embryonic stem cells from humans. Human embryonic stem cells (hESCs) are obtained from 3-5 day old embryos that remain unused after an in vitro fertilization procedure.
ES cells are grown in a culture medium where they can divide indefinitely, creating ES cell lines. Under certain conditions, ES cells can differentiate, either spontaneously into a variety of...
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Related Experiment Video

Updated: May 5, 2026

Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells
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Gene therapy, early promises, subsequent problems, and recent breakthroughs.

Saeideh Razi Soofiyani1, Behzad Baradaran, Farzaneh Lotfipour

  • 1Drug Applied Research Center, Tabriz University of Medical Sciences, Tabriz, Iran. ; Immonuology Research Center, Tabriz University of Medical Sciences, Tabriz, Iran.

Advanced Pharmaceutical Bulletin
|December 7, 2013
PubMed
Summary
This summary is machine-generated.

Gene therapy uses recombinant DNA technology for medical treatments, evolving from rare diseases to improving quality of life for various conditions. With ongoing advancements, it shows promise as a standard clinical practice within two decades.

Keywords:
Gene therapyNon-viral vectorsRecombinant DNA technologyViral vectors

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Area of Science:

  • Medicine
  • Biotechnology
  • Genetics

Background:

  • Gene therapy, a concept for half a century, became reality with recombinant DNA technology.
  • Delivery methods evolved to viral, non-viral vectors, and physical approaches for safety and efficiency.
  • Initially for severe diseases, gene therapy now targets non-life-threatening conditions impacting quality of life.

Purpose of the Study:

  • To review the progress and potential of gene therapy in clinical applications.
  • To highlight the evolution of gene delivery methods.
  • To discuss the future outlook of gene therapy in medicine.

Main Methods:

  • Review of scientific literature on gene therapy development.
  • Analysis of various gene delivery systems (viral, non-viral, physical).
  • Assessment of current and potential clinical applications of gene therapy.

Main Results:

  • Gene therapy has progressed significantly, achieving tangible successes.
  • Delivery technologies have become safer and more efficient.
  • The scope of gene therapy has expanded beyond life-threatening disorders.

Conclusions:

  • Gene therapy, though still developing, is science-based with justifiable hope for future clinical integration.
  • Continued advancements in understanding disease and biotechnology are crucial.
  • Gene therapy is anticipated to become a standard medical practice within 20 years.