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Sts Gene Therapy Final

Gene therapy is an experimental technique that uses genes to treat or prevent disease by inserting functioning genes into patients' cells instead of using drugs or surgery. It works by introducing DNA into a patient to correct effects of genetic diseases caused by mutations. The first approved gene therapy clinical trial in the US occurred in 1990. DNA is delivered via a vector carrier to the target cells. Gene therapy holds promise for treating many diseases like cancer, cystic fibrosis, and AIDS. However, there are also risks like immune reactions, viral issues, and uncertain effects on offspring.

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Arlene Poso Bala
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15K views20 pages

Sts Gene Therapy Final

Gene therapy is an experimental technique that uses genes to treat or prevent disease by inserting functioning genes into patients' cells instead of using drugs or surgery. It works by introducing DNA into a patient to correct effects of genetic diseases caused by mutations. The first approved gene therapy clinical trial in the US occurred in 1990. DNA is delivered via a vector carrier to the target cells. Gene therapy holds promise for treating many diseases like cancer, cystic fibrosis, and AIDS. However, there are also risks like immune reactions, viral issues, and uncertain effects on offspring.

Uploaded by

Arlene Poso Bala
Copyright
© © All Rights Reserved
We take content rights seriously. If you suspect this is your content, claim it here.
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Download as PPTX, PDF, TXT or read online on Scribd
  • Introduction to Gene Therapy
  • What is Gene Therapy?
  • Gene Therapy Process Diagram
  • DNA and Genetic Disease Treatment
  • Cell, Chromosome, and DNA Diagram
  • Gene vs. DNA
  • History of Gene Therapy
  • DNA Transfer Methods
  • DNA Vector Diagram
  • Types of Gene Therapy
  • Somatic Gene Therapy
  • Somatic Cell Process Diagram
  • Germline Gene Therapy
  • Pronuclear Transfer Diagram
  • Advantages and Disadvantages
  • Diseases Treated by Gene Therapy
  • Side Effects of Gene Therapy
  • Risks and Challenges
  • Importance of Gene Therapy

GENE THERAPY

SCIENCE, TECHNOLOGY & SOCIETY


PREPARED BY:
( GROUP 4 )
WHAT IS GENE THERAPY?

is an experimental technique
that uses genes to treat or
prevent disease. In the future, this
technique may allow doctors to
treat a disorder by inserting a gene
into a patient's cells instead of
using drugs or surgery.
ALSO, it is when DNA is introduced
into a patient to treat a genetic
disease. The new DNA usually
contains a functioning gene to correct
the effects of a disease-causing
mutation.
Who created gene therapy?
The first approved gene
therapy clinical research in
the US took place on 14
September 1990, at the
National Institutes of Health
(NIH), under the direction of
William French Anderson
How is DNA transfer done?

A carrier called a vector must be used


to deliver the therapeutic gene to the
patient's target cells. The vector acts
as a vehicle to carry the new DNA into
the cells of a patient with a genetic
disease.
TYPES
OF
GENE THERAPY
Somatic gene therapy
transfer of a section of DNA to
any cell of the body that doesn’t
produce sperm or eggs. Effects of
gene therapy will not be passed
onto the patient’s children.
Germline Gene Therapy
transfer of a section of DNA to cells
that produce eggs or sperm. Effects
of gene therapy will be passed onto
the patient’s children and
subsequent generations.
ADVANTAGES & DISADVANTAGES
Which diseases can be treated
with gene therapy?

Gene therapy holds promise


for treating a wide range
of diseases, such as cancer,
cystic fibrosis, heart disease,
diabetes, hemophilia and AIDS
Side effects of gene therapy

the patient's immune system


may react to the foreign vector,
causing fever, severe chills (called
rigors), drop in blood pressure,
nausea, vomiting, and headache.
These symptoms typically resolve
within 24-48 hours of the infusion.
•DNA mutations
•Immune response
•Viral spread
Reversion of the virus
to its original form
•Risk to offspring
•High cost
Why Gene Therapy Is Important?

The goal of gene therapy is to cure or


slowdown a genetic disease by repairing
the damaged gene responsible for
the disease. To achieve this goal, gene
therapy requires the technology to insert
the missing “normal” gene into the DNA
of the patient's cells.

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