GENE THERAPY

• Terminology
• Origin
Slide 3-5 • Purpose
• When & where it started
• Who invented Slide 6-7
• How it works?
Slide 8- (Components, Elements,
Procedures)
• Contributions to Science
and Technology & Society Slide 9-10

• Succesful operation
Slide 11- 15

• Advantages & Disadvantages

Slide 16-19
to Science, Technology &
Society
• Gene comes from the greek word “genos” or “gen” which means race,
kind, family or birth.
• Therapy- treatment, to relieve or heal.
• Human gene therapy seeks to modify or manipulate the expression of
a gene or to alter the biological properties of living cells for therapeutic
use.
• Gene therapy replaces a faulty gene or adds a new gene in an attempt
to cure disease or improve your body's ability to fight disease.
• The concept of gene therapy arose during 1960’s and early 1070’s whilst
the development of genetically marked cells lines and the clarification of
mechanisms of cell transformation by the papaovaviruses polyoma and
SV40 was in progress.
• First approved gene therapy clinical research was done in United States
took place on September 14, 1990 at the National Institues of Health
(NIH), under the direction of William French Anderson.
Gene therapy is a technique that modifies a person’s genes to treat or
cure disease. Gene therapies can work by several mechanisms:

v Replacing a disease-causing gene with a healthy copy of the gene

v Inactivating a disease-causing gene that is not functioning properly
v Introducing a new or modified gene into the body to help treat a
disease
William French Anderson
Anderson was a pioneer of gene therapy. He was the
first to use this innovative method in an attempt to
correct a human genetic disorder. In September 1990,
he infused gene-corrected T-lymphocytes into a child
with severe combined immunodeficiency (SCID), a rare
genetic disorder caused by an adenosine deaminase-
defective gene. That courageous step opened the door
for numerous trials of gene therapy for different
conditions, particularly cancer.
• The first gene therapy trial launched in
1990. A four-year-old girl named Ashanthi
DeSilva underwent a 12-day treatment for a
rare genetic disease known as severe
combined immunodeficiency. DeSilva
lacked a key enzyme called adenosine
deaminase (ADA), which left her immune
system crippled and put her at constant risk
of contracting an infection that could kill her.
Ashanthi DeSilva
 Replacing a disease-causing gene with a healthy copy of the gene.
Inactivating a disease-causing gene that is not functioning properly.

https://www.exploregenetherapy.com/how-gene-replacement-therapy-works
 • A genetic retina disorder that causes
visual impairement beginning in infancy

• On August 2009, a clinical trial involving

three (3) patients shows positive result for
healthy and improved vision.

https://www.exploregenetherapy.com/how-gene-replacement-therapy-works
 • A mutation in the Dystrophin gene affects both
skeletal and muscles.
• Most commonly inherited myopathy in childhood
• Typically affects thighs & pelvis followed by arms
• Trouble standing up
• Most are unable to walk by the age of 12
• Gene therapy trials on mice and dogs have shown
positive result.

https://www.exploregenetherapy.com/how-gene-replacement-therapy-works
 • A mutation in the Dystrophin gene affects
both skeletal and cardiac muslces
• Most commonly inherited myopathy in
childhood
• Gene therapy trials on mice and dogs have
shown positive result.

https://www.exploregenetherapy.com/how-gene-replacement-therapy-works
 • Type of cancer where the bone marrow makes
too many imature lymphocytes and affects the
bood chemistry
• FDA has approved two (2) CAR-T therapies by
2017
• Patients shows high remission rates after
treatment

https://www.exploregenetherapy.com/how-gene-replacement-therapy-works
 1.New knowledge which serves as a direct source of ideas for new technological possibilities.
2.Source of tools and techniques for more efficient engineering design and a knowledge
base for evaluation of feasibility of designs.
3.Research instrumentation, laboratory techniques and analytical methods used in research
that eventually find their way into design or industrial practices, often through intermediate
disciplines.
4.Practice of research as a source for development and assimilation of new human skills and
capabilities eventually useful for technology.
5.Creation of a knowledge base that becomes increasingly important in the assessment of
technology in terms of its wider social and environmental impacts.

https://www.exploregenetherapy.com/how-gene-replacement-therapy-works
 vGene Therapy has the potential to eliminate and prevent hereditary
diseases.
. vGene therapy can create permanent results.
vTherapy may only need to be delivered once.
vGene therapy does not just apply to human treatment options.
• Immune response
The body's immune system may see the newly introduced viruses as intruders and attack them resulting in
inflammation, toxicity, as well as organ failure in severe cases.
• Viral spread.
viruses can affect more than one type of cell, it is possible that the viral vectors may infect cells beyond just
those containing mutated or missing genes. If this happens, healthy cells may be damaged and cause other
illness or diseases.
• Side effects.
After initially receiving a type of gene therapy, the immune system may react to the foreign
vector. Symptoms of a reaction may include fever, drop in blood pressure, nausea, vomiting, and headache
• DNA Mutation
The new gene might be inserted in the wrong location in the DNA, which might cause harmful mutations to
the DNA.
Gene therapy is an expensive procedure.
CABATINGAN, LIBECCA

CRISOSTOMO, KRISHNA KAYE

GELLA, BEA TRICIA

MEDTIMBANG, NUR ALEAH

PACIFICAR, JON

SAWABE, JUL-ANI

VELASCO, RICO
REFRENCES

https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/what-gene-therapy

https://www.mayoclinic.org/tests-procedures/gene-therapy/about/pac-20384619

https://pubmed.ncbi.nlm.nih.gov/1303270/#:~:text=The%20concepts%20of%20gene%20therapy,and%20SV40%20was
%20in%20progress.

https://www.whatisbiotechnology.org/index.php/science/summary/gene-therapy/

https://www.labiotech.eu/in-depth/gene-therapy-history/

https://www.youtube.com/watch?v=BxEoX6TkitY&t=1s&ab_channel=ASGCT
Thank you !