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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...
What is Genetic Engineering?00:49

What is Genetic Engineering?

Overview
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.
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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Engineering immune tolerance for AAV gene therapy: The Norse way.

Molecular therapy : the journal of the American Society of Gene Therapy·2026
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Hemophilia A gene therapy: Moving to the next chapter.

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Transient prophylactic immunosuppression with abatacept or dasatinib prevents immune responses in AAV gene transfer.

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Current Status of Clinical Gene Therapy for Hemophilia and Globin Disorders.

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Factor VIII originates primarily from anatomically distinct subsets of liver sinusoidal endothelial cells.

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Deaths in gene therapy of Duchenne muscular dystrophy and other diseases: Underlying mechanisms and mitigating strategies.

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Related Experiment Video

Updated: May 23, 2026

Subretinal Injection of Gene Therapy Vectors and Stem Cells in the Perinatal Mouse Eye
05:09

Subretinal Injection of Gene Therapy Vectors and Stem Cells in the Perinatal Mouse Eye

Published on: November 25, 2012

Seeing eye to eye with gene therapy

Roland W Herzog

    Molecular Therapy : the Journal of the American Society of Gene Therapy
    |April 5, 2012
    PubMed
    Summary

    No abstract available in PubMed .

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