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Related Concept Videos

Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

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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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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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What is Genetic Engineering?00:49

What is Genetic Engineering?

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Combination Therapies and Personalized Medicine02:50

Combination Therapies and Personalized Medicine

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Combining two or more treatment methods increases the life span of cancer patients while reducing damage to vital organs or tissue from the overuse of a single treatment. Combination therapy also targets different cancer-inducing pathways, thus reducing the chances of developing resistance to treatment.
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...
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Targeted Cancer Therapies02:57

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The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
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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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Related Experiment Videos

Gene therapy---perspectives and promises.

Y L Kwong1

  • 1Department of Medicine, The University of Hong Kong, Queen Mary Hospital, Pokfulam, Hong Kong.

Hong Kong Medical Journal = Xianggang Yi Xue Za Zhi
|June 1, 1997
PubMed
Summary

Gene therapy shows promise for genetic disorders and cancer, with ongoing research improving efficiency and specificity. Despite challenges, compelling evidence suggests gene therapy will become a key treatment for genetic diseases.

Area of Science:

  • Biotechnology
  • Genetics
  • Medicine

Background:

  • Gene therapy is an emerging treatment for genetic disorders.
  • In vivo gene therapy has demonstrated feasibility and biological responses in various human diseases.
  • Research is increasingly focusing on gene therapy for cancer treatment.

Purpose of the Study:

  • To review the current state and future potential of gene therapy.
  • To highlight advancements in gene transfer efficiency and specificity.
  • To discuss the evolving applications of gene therapy in human diseases.

Main Methods:

  • Review of basic laboratory investigations and clinical trials in gene therapy.
  • Analysis of research focusing on improving gene transfer methods.

Related Experiment Videos

  • Examination of the shift in gene therapy applications towards cancer treatment.

    • Main Results:

    • Gene therapy is feasible in vivo and elicits biological responses.
    • Improvements in gene transfer efficiency and specificity are key research goals.
    • Gene therapy offers insights into disease mechanisms and is expanding into cancer treatment.

    Conclusions:

    • Gene therapy is a promising modality for inherited and acquired genetic disorders.
    • Despite existing limitations, compelling evidence supports gene therapy's efficacy.
    • Gene therapy is expected to overcome current challenges and become a significant treatment option for genetic diseases.