Professional Documents
Culture Documents
Presented by:
Nurul Miza Shasheiha binti Abdul Mutalib -5089-
Wan Fathiah Nasuha binti Wan Nudri -5117-
Nurul Husna binti Muryadi -5088-
Nur Amalin Shafirah binti
Nur Farzana Sahirah binti Ariffuddin
WHAT IS GENE THERAPY ?
• Definiton: an experimental technique for correcting defective
genes that are responsible for disease development
• The most common form of gene therapy involves inserting a
normal gene to replace an abnormal gene
• Other approaches used:
Replacing a mutated gene that causes disease with a
healthy copy of the gene.
Inactivating, or “knocking out,” a mutated gene that is
functioning improperly.
Introducing a new gene into the body to help fight a disease.
• Researchers are studying gene therapy for a number of
diseases, such as
Severe combined immuno-deficiencies (SCID)
Hemophilia
Parkinson's disease
Cancer
HIV
HISTORY AND DEVELOPMENT
OF GENE THERAPY
• 1960: The concepts of Gene Therapy was introduced
• 1970: Friedmann and Roblin author of a paper in Science titled "Gene
therapy for human genetic disease?” cite the first attempt to perform
gene therapy
• 1990:
The first approved gene therapy case at the National Institute of
Health, U.K. It was performed on a four year old girl named
Ashanti DaSilva. It was a treatment for a genetic defect that left
her with an immune system deficiency
New gene therapy approach repairs errors in messenger RNA
derived from defective genes. This technique has the potential to
treat the blood disorder Thalassaemia, Cystic fibrosis, and some
cancers
Sickle cell disease is successfully treated in mice
• 1992: Doctor Claudio Bordignon working at the Vita-Salute San Raffaele
University, Milan, Italy performed the first procedure of gene therapy using
hematopoietic stem cells as vectors to deliver genes intended to correct
hereditary diseases
• 1999: Death of Jesse Gelsinger in a gene-therapy experiment resulted in a
significant setback to gene therapy research in the United States
• 2006: Scientists at the National Institutes of Health (Bethesda, Maryland) have
successfully treated metastatic melanoma in two patients. This study constitutes
one of the first demonstrations that gene therapy can be effective in treating
cancer.
• 2007- 2011: Research is still ongoing and the number of diseases that has been
treated successfully by gene therapy increases.
Retinal disease
Colour blindness
Adrenoleukodystrophy
• 2011: Medical community accepted that it can cure HIV as in 2008, Gero Hutter
has cured a man from HIV using gene therapy
Result in permanent changes.
Potential for offering a permanent therapeutic
effect for all who inherit the target gene.
Possibility of eliminating some diseases from a
particular family.
Also raises controversy:
Some people view this type of therapy as
unnatural, and liken it to "playing God”.
Others have concerns about the technical
aspects.
Affects only the targeted cells in the
patient, and is not passed to future
generations.
Short-lived because the cells of most tissues
ultimately die and are replaced by new
cells.
Transporting the gene to the target cells or
tissue is also problematic.
Appropriate and acceptable for many
disorders, including cystic fibrosis, muscular
dystrophy, cancer, and certain infectious
diseases.
BASIC PROCESS OF
GENE THERAPY
1. VIRAL VECTOR
2. NON VIRAL VECTOR
GT utilizes the delivery of DNA into cells, which can be
accomplished by a number of methods.
VIRAL VECTOR
Virus bind to their hosts and introduce their genetic
material into the host cell.
VIRUS
Many GT clinical trials rely on retroviruses or
adenoviruses to deliver the desired gene.
TYPES OF VIRUS
Are a tool commonly used by molecular biologists to
deliver genetic material into cells.
VIRAL VECTOR
Methods of non-viral gene delivery have also been
explored using physical (carrier-free gene delivery)
and chemical approaches (synthetic vector-based
gene delivery).
PHYSICAL METHOD
The simplest method of non-viral transfection.
Clinical trials carried out of intramuscular injection
of a naked DNA plasmid have occurred with some
success; however, the expression has been very low
in comparison to other methods of transfection.
I. NAKED DNA
This success, however, does not compare to that of
the other methods, leading to research into more
efficient methods for delivery of the naked DNA
such as electroporation and the use of a "gene gun",
which shoots DNA coated gold particles into the cell
using high pressure gas.
CHEMICAL METHODS
THAT ENHANCE THE
DELIVERY OF GENE THERAPY
-lipoplexes & polyplexes-
DNA must be protected from damage & its entry
into the cell must be facilitated
Plasmid DNA can be covered with lipids in an
organized structure like a micelle or a
liposomecomplexed with DNA it is called a
lipoplex
3 types of lipids:
anionic (negatively charged)
neutral
cationic (positively charged)
LIPOPLEXES
Initially, anionic and neutral lipids :
-were used for the construction of lipoplexes for
synthetic vectors.
-but,there is little toxicity associated with them,
-they are compatible with body fluids
-there was a possibility of adapting them to be
tissue specific
-they are complicated turned to the cationic
versions.
POLYPLEXES
SUCCESS CASES OF GENE
THERAPHY
PARKINSON’S
BLINDNESS
DISEASE
GENE THERAPY CURES
BLINDNESS
Cure blindness of inherited condition
Leber’s conginetal amaurosis
- inherited disease caused by an
abnormality in a gene called RPE65.
- The condition appears at birth or in the first
few months of life and causes progressive
worse and loss of vision.
HOW IT WORKS??
used harmless viruses
enable access to the cells beneath the
retinas of patients
By using a very fine needle
-safe in an extremely fragile tissue and can
improve vision in a condition previously
considered wholly untreatable.
• http://www.youtube.com/watch?v=d_YJZn-ft_Q
SUCCESS CASES OF GENE
THERAPHY
PARKINSON’S
BLINDNESS
DISEASE
GENE THERAPY REDUCES
PARKINSON’S DISEASE SYMPTOMS