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E X P LO R I NG

GENE THERAPY WHAT IS GENE THERAPY?

Each person inherits features from his or her parents in the form of genes1.

When a gene does not work properly, the gene is said to be malfunctioning and may cause a genetic disease1.

Genes are made up of a molecule called DNA (deoxyribonucleic acid)1.

Gene therapy uses functioning genes as medicine to help correct a genetic defect2.

HOW DOES GENE THERAPY WORK? 2

4

In a lab, a functioning copy of the gene is inserted into the DNA of the stem cells, referred to as transduction, using a modified virus.

3

1

Chemotherapy is given to make room for the modified stem cells. The modified stem cells are then transplanted back into the body.

Stem cells are collected from the blood.

The goal is for modified stem cells to become a permanent source of blood cells that grow and produce new cells with the functioning copy of the gene.

WHY GENE THERAPY NOW?

In 2012, the first gene therapy was approved in Europe4.

Significant advances have been made in gene transfer technology, safety and how the transfer vehicles are manufactured.

Long term safety and efficacy has been observed for more than 5 years and positive data is growing.

30 YEARS,

~2,000

Gene therapy has been studied as a potential treatment for genetic disease2.

clinical trials have been conducted using different types of gene therapy in cancers, blood diseases, central nervous system disorders and immune system diseases2,3,5.

For

THE GOAL

transform the treatment of genetic diseases BY PROVIDING FUNCTIONING COPIES OF THE MALFUNCTIONING GENES.

Sources: 1. www.ornl.gov/sci/techresources/Human_Genome/medicine/ genetherapy.shtml (Accessed Dec. 4, 2012). 2. Sheridan, C., 2011. Gene therapy finds its niche. Nature Biotechnology, 29(2), pp. 121–8. 3. Kay, M. A., 2011. State-of-the-art gene-based therapies: the road ahead. Nature, 12(5), pp. 316-28. 4. www.uniqure.com/news/167/189/uniQure-s-Glybera-First-Gene-Therapy-Approved-by-European-Commission.html (Accessed Feb. 18, 2013). 5. Kohn, D.B. & Candotti, F., 2009. Gene therapy fulfilling its promise. The New England Journal of Medicine, 360(5), pp. 518–21.

Profile for Thalassaemia International Federation (TIF)

Gene Therapy Fact Sheet  

Gene Therapy Fact Sheet  

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