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Related Concept Videos

Gene Therapy00:59

Gene Therapy

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 inserted. The...
Gene Therapy00:59

Gene Therapy

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 inserted. The...
Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

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.
Pharmacogenomics: Identification of New Drug Targets01:29

Pharmacogenomics: Identification of New Drug Targets

Advances in genomics have profoundly influenced drug discovery by increasing both the speed and accuracy of pharmaceutical development. Pharmacogenomics, which examines how genetic variation influences drug response, facilitates the identification of novel therapeutic targets and enables patient stratification for personalized treatment. These strategies contribute to improved drug efficacy, minimized adverse effects, and more efficient clinical trial design.Mapping genetic differences...
Targeted Cancer Therapies02:57

Targeted Cancer Therapies

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.
There are several types of targeted therapies against specific...
Targeted Cancer Therapies02:57

Targeted Cancer Therapies

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.
There are several types of targeted therapies against specific...

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Related Experiment Video

Updated: Jun 4, 2026

Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells
09:37

Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells

Published on: August 25, 2021

Gene therapy finds its niche.

Cormac Sheridan

    Nature Biotechnology
    |February 9, 2011
    PubMed
    Summary

    Gene therapy offers new hope for treating severe genetic diseases that resist other treatments. This approach is now ready to provide significant therapeutic benefits.

    Area of Science:

    • Biotechnology
    • Medical Genetics
    • Molecular Biology

    Background:

    • Genetic diseases present significant challenges in treatment.
    • Many debilitating genetic conditions lack effective therapeutic options.

    Purpose of the Study:

    • To highlight the emerging role of gene therapy in treating genetic disorders.
    • To underscore the potential of gene therapy for previously intractable diseases.

    Main Methods:

    • Review of current gene therapy approaches.
    • Analysis of clinical trial data for genetic diseases.

    Main Results:

    • Gene therapy demonstrates significant promise for treating debilitating genetic diseases.
    • This therapeutic modality is effective for conditions resistant to conventional treatments.

    More Related Videos

    Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells
    11:16

    Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells

    Published on: February 15, 2019

    Related Experiment Videos

    Last Updated: Jun 4, 2026

    Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells
    09:37

    Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells

    Published on: August 25, 2021

    Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells
    11:16

    Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells

    Published on: February 15, 2019

    Conclusions:

    • Gene therapy is poised to become a major therapeutic strategy for genetic diseases.
    • The advancement of gene therapy offers new hope for patients with severe genetic conditions.