1 Slide: Chapter 11: Gene Therapy

Chapter 11: Gene Therapy
1st slide
Learning Objectives:
At the end of this chapter, the students should be able to:
1. Describe gene therapy and its various forms
2. Discuss the prevalence of gene therapy in daily life, and
3. Explore the opportunities that may be opened by gene therapy in the future
2nd slide
What is Gene Therapy?
Gene therapy is the use of genetic modification to treat human disease.
(INSERT GIF/PIC OF GENE THERAPY)
A sick patient harbors a bad version of a gene, gene therapy delivers a good
version of that gene into cells within the sick patient. The majority of diseases treated
with gene therapy are inherited genetic diseases (Down syndrome, Cystic Fibrosis).
However, you can also use gene therapy to treat certain cancers and viral infections.
3rd slide
2 TYPES OF GENE THERAPY
There are two different types of gene therapy depending on which types of cells
are treated:
4th slide
 Somatic gene therapy: (INSERT GIF SOMATIC GENE THERAPY)
- Transfer of a section of DNA to any cell of the body that doesn’t produce sperm
or eggs (somatic cells).
- Effects of gene therapy will not be passed onto the patient’s children.
- Aim to cure a disease only in the patient, not in the patient's descendants .
5th slide
 Germline gene therapy: (INSERT GIF GERMLINE GENE THERAPY)
- Transfer of a section of DNA to cells that produce eggs or sperm (reproductive

cells).
- Effects of gene therapy will be passed onto the patient’s children and subsequent
generations.
- allows for the correction of disease-causing gene variants that are certain to be
passed down from generation to generation
6th slide
Prevalence of Gene Therapy
What we should know about the stem cell treatment in the PH?
(INSERT GIF OF STEM CELL THERAPY)
-a type of intervention strategy that introduces new adult stem cells into damaged tissue
in order to treat disease or injury, including cancer and HIV as being alleged in news
reports.
(further infos nasa book)
Hospitals, medical centers and wellness centers offering stem cell therapy for medical
and aesthetic purposes have proliferated rapidly in the Philippines.
7th slide
The Future of Gene Therapy
 The Human Genome Project
- Sequencing and mapping virtually all of the 25,000-30,000 genes in the cell.
- Provide new strategies to diagnose, treat, cure and possibly prevent human
diseases.
- “The future revolution of medicine”
- Hopes are high for its role in curing and preventing childhood diseases
- May be possible to treat an unborn child for a genetic disease even before
symptoms appear
To cure genetic diseases, scientists must first determine which gene or set of genes causes
each disease. The Human Genome Project and other international efforts have completed the initial
work of sequencing and mapping virtually all of the 25,000 genes in the human cell. This research
will provide new strategies to diagnose, treat, cure, and possibly prevent human diseases.
Although this information will help scientists determine the genetic basis of many diseases, it will be
a long time before diseases actually can be treated through gene therapy.
Gene therapy's potential to revolutionize medicine in the future is exciting, and hopes are high for its
role in curing and preventing childhood diseases. One day it may be possible to treat an unborn child
for a genetic disease even before symptoms appear.
Scientists hope that the human genome mapping will help lead to cures for many diseases and that
successful clinical trials will create new opportunities. For now, however, it's a wait-and-see situation,
calling for cautious optimism.
Other readings about gene therapy….. (not included in the ppt)
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. Researchers are testing several
approaches to gene therapy, including:
 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.
Although gene therapy is a promising treatment option for a number of diseases

(including inherited disorders, some types of cancer, and certain viral infections), the
technique remains risky and is still under study to make sure that it will be safe and
effective. Gene therapy is currently being tested only for diseases that have no other
cures.
Gene therapy is an experimental technique that uses genes to treat or prevent

disease.

cures.
Gene therapy 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.
 Gene therapy uses sections of DNA? (usually genes?) to treat or prevent disease.

 The DNA is carefully selected to correct the effect of a mutated gene that is
causing disease.
 The technique was first developed in 1972 but has, so far, had limited success in
treating human diseases.
 Gene therapy may be a promising treatment option for some genetic diseases?,
including muscular dystrophy? and cystic fibrosis?.
There are two different types of gene therapy depending on which types of cells are
treated:
 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.
Gene therapy is an experimental technique that uses genes to treat or prevent disease.

cures.
Gene therapies are gaining momentum as promising early successes in clinical studies
accumulate and examples of regulatory approval for licensing increase. Investigators are
advancing with cautious optimism that effective, durable, and safe therapies will provide benefit
to patients—not only those with single-gene disorders but those with complex acquired diseases
as well. While the strategies being translated from the lab to the clinic are numerous, this review
focuses on the clinical research that has forged the gene therapy field as it currently stands.
Last slide
References (included in ppt)
https://www.yourgenome.org/facts/what-is-gene-therapy
https://ghr.nlm.nih.gov/primer/therapy/genetherapy
https://kidshealth.org/en/parents/gene-therapy.html#:~:text=Germline%20gene%20therapy%2C
%20which%20involves,offspring%20had%20the%20new%20gene.
https://www.annualreviews.org/doi/abs/10.1146/annurev-med-012017-043332?journalCode=med

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Chapter 11: Gene Therapy

(INSERT GIF/PIC OF GENE THERAPY)

2 TYPES OF GENE THERAPY

 Somatic gene therapy: (INSERT GIF SOMATIC GENE THERAPY)

 Germline gene therapy: (INSERT GIF GERMLINE GENE THERAPY)

- Transfer of a section of DNA to cells that produce eggs or sperm (reproductive

Prevalence of Gene Therapy

(INSERT GIF OF STEM CELL THERAPY)

(further infos nasa book)

The Future of Gene Therapy

 The Human Genome Project

Other readings about gene therapy….. (not included in the ppt)

Although gene therapy is a promising treatment option for a number of diseases

Gene therapy is an experimental technique that uses genes to treat or prevent

Although gene therapy is a promising treatment option for a number of diseases

 Gene therapy uses sections of DNA? (usually genes?) to treat or prevent disease.

Although gene therapy is a promising treatment option for a number of diseases

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