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Gene Therapy

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 Therapy

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?

What is Genetic Engineering?

Targeted Cancer Therapies

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 Therapies

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...

Microorganisms in Medicine and Therapeutics

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.

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Related Experiment Video

Updated: May 11, 2026

Linear Amplification Mediated PCR – Localization of Genetic Elements and Characterization of Unknown Flanking DNA

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[Gene therapy: where do we stand?].

Marina Cavazzana-Calvo¹

¹U768 Inserm, Paris, France. m.cavazzana@nck.aphp.fr

La Revue Du Praticien

|May 21, 2013

Summary

Gene therapy offers targeted molecular medicine by adding corrected genes to somatic cells for treating inherited diseases and cancers. Recent successes show clinical benefits, but preventing severe adverse events remains crucial for wider application.

Area of Science:

Molecular medicine
Genetics
Biotechnology

Context:

Gene therapy, a molecular medicine approach, involves introducing corrected genes into somatic cells.
Initially explored for cancer treatment in the 1990s, it has faced challenges and achieved successes.
Recent advancements have demonstrated significant clinical benefits for various conditions.

Purpose:

To provide targeted therapies for acquired or inherited diseases.
To explore the potential of gene addition for disease cure or alleviation.
To assess the progress and challenges in gene therapy applications.

Summary:

Gene therapy utilizes gene addition to correct genetic defects in somatic cells.
It has shown promise in treating hematopoietic diseases, retinal disorders, and certain cancers.

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Overcoming obstacles and ensuring stable prevention of adverse events are key to its advancement.

Impact:

Gene therapy expands treatment options for a growing number of genetic and acquired diseases.
Successful clinical applications are widening, contingent on managing severe adverse events.
This therapeutic strategy holds significant potential for future medical interventions.