Gene Therapy

Intended Learning
Outcomes:

describe gene therapy and its various forms;

and
assess the issues, potential benefits and
detriments to global health
 Your genetics load the gun.
Your lifestyle pulls the trigger.

- Mehmet oz
Genome is a complete set of the genes in a cell
or organism.

Chromosomes of the human genome

What is gene therapy?

Gene therapy is a technique that

modifies a person’s genes to
treat or cure disease. Gene
therapy products are being
studied to treat diseases
including cancer, genetic
diseases, and infectious diseases
(fda.gov 2018).
Gene therapies can work by several
mechanisms (fda.gov 2018):

• Replacing a disease-causing gene with a

healthy copy of the gene
• Inactivating a disease-causing gene that
is not functioning properly
• Introducing a new or modified gene into
the body to help treat a disease
 Types of Gene therapy products
1. Plasmid DNA. Circular DNA molecules can
be engineered genetically to bring therapeutic
genes into human cells.
 Types of Gene therapy products
2. Viral vectors. Once viruses have been
modified to remove their ability to cause
infectious disease, these modified viruses can
be used as vectors to carry therapeutic genes
into human cells.
 Types of Gene therapy products
3. Bacterial vectors. Bacteria can be modified
genetically to prevent them from causing
infectious disease and then used as vectors to
carry therapeutic genes into human tissues.
 Types of Gene therapy products
4. Human gene
editing
technology. The
goal of gene
editing is to
disrupt harmful
genes or to
repair mutated
genes.
 Types of Gene therapy products
5. Patient-derived cellular gene therapy products.
Cells are removed from the patient, genetically
modified (often using a viral vector) and then
returned to the patient.
 Two Types of Gene Therapy

1. Somatic gene 2. Germ-line gene

therapy- involves the therapy- involves the
manipulation of genetic modification
genes in cells that will of germ cells or the
be useful to the origin cells that will
patient but not pass the change on to
inherited to the next the next generation
generation (Your Genome, 2017).
(Nimsergern, 1988)
 Two
Types of
Gene
Therapy
 Gene Therapy Basics

https://www.youtube.com/watch?v=BxEoX6TkitY
 Risks
and
Benefits
 Benefits
1. It offers cure to several diseases like cancer,
Parkinson’s disease, AIDS, asthma, diabetes,
heart diseases as well as hereditary diseases.
2. It can replace defective cells.
3. It has a promising potential not only in the field
of medicine but in some related fields like
agriculture.
 Disadvantages
1. Modifying organisms means modifying its
capabilities.
2. Viral vectors could recover its ability to cause
disease.
3. It can damage the gene pool resulting to cause
disease.
4. High cost.
5. Ethical issues.
References
• Bautista, D. H. et al. (2018). Science, Technology, and Society.
Quezon City: MaxCor Publishing House, Inc.
• Serafica, et. al. (2018). Science, Technology, and Society. Quezon
City: Rex Printing Company, Inc.
• https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy
products/what-gene-therapy
Thank you