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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...

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...

Satellite Stem Cells and Muscular Dystrophy

Satellite Stem Cells and Muscular Dystrophy

Satellite stem cells or myosatellite cells are quiescent stem cells that Alexander Mauro first identified in 1961. These cells are located between the sarcolemma, the plasma membrane of muscle fibers, and the basal lamina, the connective tissue sheath covering it. These mononucleated cells are activated in response to muscle injury, can transform into myoblasts, and may form or repair muscle fibers. Myosatellite cells can provide additional myonuclei for muscle regeneration or return to a...

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Related Experiment Video

Updated: Jun 17, 2026

An In Vitro Model for the Study of Cellular Pathophysiology in Globoid Cell Leukodystrophy

An In Vitro Model for the Study of Cellular Pathophysiology in Globoid Cell Leukodystrophy

Published on: October 21, 2014

Gene therapy in metachromatic leukodystrophy.

C Sevin¹, N Cartier-Lacave, P Aubourg

¹French Institute for Health and Medical Research, Paris Descartes University and Department of Pediatric Neurology, Hôpital Saint-Vincent de Paul, Paris, France.

International Journal of Clinical Pharmacology and Therapeutics

|December 31, 2009

Summary

Metachromatic leukodystrophy (MLD) is a rare genetic disorder. This review explores gene therapy using hematopoietic stem cells or direct brain gene transfer as potential treatments for MLD patients.

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Area of Science:

Neuroscience
Genetics
Biochemistry

Background:

Metachromatic leukodystrophy (MLD) is a lysosomal storage disease.
It stems from arylsulfatase A enzyme deficiency.
This deficiency causes harmful sulfatide buildup in the brain and nervous system.

Purpose of the Study:

To review the therapeutic potential of gene therapy for MLD.
Focuses on hematopoietic stem cell gene therapy and intracerebral gene transfer.

Main Methods:

Review of existing literature on MLD.
Analysis of gene therapy approaches for lysosomal storage diseases.

Main Results:

Hematopoietic stem cell gene therapy shows promise for MLD treatment.
Intracerebral gene transfer is another viable therapeutic strategy.

Conclusions:

Gene therapy offers a potential treatment avenue for MLD.
Further research is needed to optimize these gene therapy approaches for MLD.