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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.