Download - Folleto Juan Felipe Giraldo Aristizábal
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Juan Felipe Giraldo Aristizábal
Medicine Student
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FOLDING
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FOLDING
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INTRODUCTIO
N
• Gene therapy is the
use of DNA as a
pharmacological
agent in order to alter
certain parts of the
genome of a life
being that are
mutated or
dysfunctional, to
repair the protein non
efficient synthesis.
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Gene Therapy Using Lentivirus to Treat Wiskott-Aldrich
Syndrome Promising
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Gene Therapy Using Lentivirus to Treat Wiskott-
Aldrich Syndrome Promising
• The Wiskott-Aldrich Syndrome is
caused by a mutation in a gene
that encodes the protein WASP.
• The most often used therapy is
a bone marrow or stem cell
transplant from a matching
donor, and can be curative
mostly for those whose donor is
strongly matched.
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Gene Therapy Using Lentivirus to Treat Wiskott-
Aldrich Syndrome Promising
An alternative is to use a form of retrovirus using gene therapy in the
laboratory and apply it to the blood cells previously obtained from the patient
so the retrovirus can correct the defect, and then give the corrected cells
back to the patient.
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Gene Therapy Using Lentivirus to Treat Wiskott-
Aldrich Syndrome Promising
This approach has been successful in
a number of diseases, including who
had Wiskott-Aldrich Syndrome.
However, over the long term, some
patients with immune deficiencies,
including those with Wiskott-Aldrich
Syndrome, developed blood cancers.
In this new research, the team used
a partially inactivated lentivirus to
take the normal gene into the cell,
while reducing the risk of the gene
inserting next to a cancer-
promoting gene.
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This type of treatment instead of
previous treatments using gene
therapy with retroviruses is an
amazing option for this type of
patients, considering the lower risk
of developing a cancer or other
collateral diseases. This gene
therapy also allows the patients to
have a more normal and
comfortable life increasing their life
quality.
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Gene Therapy Cures a Severe Pediatric
Neurodegenerative Disease in Animal Models
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Gene Therapy Cures a Severe Pediatric
Neurodegenerative Disease in Animal Models
• Sanfilippo Syndrome type A, is
a neurodegenerative disease
caused by mutations in the
gene that encodes the enzyme
sulfamidase.
• The accumulation of
glycosaminoglicans in the cells
causes neuroinflammation
and organ dysfunction.
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Gene Therapy Cures a Severe Pediatric
Neurodegenerative Disease in Animal Models
A team of researchers of the
UAB's Centre for Animal
Biotechnology and Gene
Therapy (CBATEG), has
developed a gene therapy
treatment that cures this disease
in animal models, with pre-
clinical studies in mice and dogs.
The treatment consists of a single
surgical intervention in which an
adenoassociated viral vector, which is
completely harmless, is injected into the
cerebrospinal fluid, the liquid that
surrounds the brain and the spinal cord.
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Gene Therapy Cures a Severe Pediatric
Neurodegenerative Disease in Animal Models
Once the enzyme's activity is
restored, glycosaminoglican levels
return to normal for life, their
accumulation in cells disappears,
along with the neuroinflammation
and dysfunctions of the brain and
other affected organs, and the
animal's behaviour and its life
expectancy return to normal.
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It’s overwhelming how the gene
therapy has gone so far to the point of
cure once and for all a
neurodegenerative disease like
Sanfilippo Syndrome type A. Although
the treatment has not being studied in
humans, the fact that it has been
successful in animal models leaves us
with great expectations of what’s
coming next.
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• Baylor College of Medicine (2013, July11). Gene therapy using lentivirus to treatWiskott-Aldrich Syndromepromising. ScienceDaily. Retrieved July22, 2013, fromhttp://www.sciencedaily.com/releases/2013/07/130711142403.htm
• Universitat Autònoma de Barcelona (2013, July 2). Gene therapy cures a severepediatric neurodegenerative disease in animal models.ScienceDaily. RetrievedJuly 22, 2013, fromhttp://www.sciencedaily.com/releases/2013/07/130702100344.htm
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THANKS
!