Lesson 4

Gene Therapy (A promising future to disease treatment)
- The introduction of genes to existing cells to prevent or cure a wide range of diseases.
- A technique in correcting defective genes responsible for disease development.
- The earliest method of gene therapy, often called gene transfer or gene addition
Types of Gene Therapy

Somatic Cell Gene Therapy
- Therapeutic Genes transferred into somatic cells.
- In Vivo( inside your body)
- Scienctist put the vector carrying the new genes directly into your body via injection or
intravenus (IV) infusion.
Germ Line Gene Therapy

- Therapeutic Genes transferred into the germ cell
- Ex Vivo ( outside your body)
- Scientist extract cells from your body and introduce them to the vector inside a petri dish,then
your altered cell are returned to your body when they hopefully multiply.
What can it treat?

1. Inherited vision loss
2. Blood disorders
3. Spinal muscular atrophy ( SMA)
4. Cerebral adrenoleukodystrophy ( CALD)
5. Cancer
Genome Editing
- Introduces molecular tools to change the existing DNA in the cell.
- Genome editing is being studied to:
● Fix a genetic alteration underlying a disorder, so the gene can function properly.
● Turn on a gene to help fight a disease.
● Turn off a gene that is functioning improperly.
● Remove a piece of DNA that is impairing gene function and causing disease.
How does gene therapy work?

- Gene therapy works by altering the genetic code to recover the functions of critical proteins
- Certain viruses are used as vectors because they can deliver the material by infecting the cell.
- The vector can be injected or given intravenously (by IV) directly into a specific tissue in the
body, where it is taken up by individual cells.
- Alternately, a sample of the patient's cells can be removed and exposed to the vector in a
laboratory setting.
-The cells containing the vector are then returned to the patient. If the treatment is successful,
the new gene delivered by the vector willmake a functioning protein or the editing molecules will
correct a DNA error and restore protein function.
Gene therapy can compensate for genetic alterations in a couple different ways.
- Gene transfer therapy introduces new genetic material into cells. If an altered gene causes a
necessary protein to be faulty or missing, gene transfer therapy can introduce a normal copy of
the gene to recover the function of the protein. Alternatively, the therapy can introduce a
different gene that provides instructions for a protein that helps the cell function normally,
despite the genetic alteration.
- Genome editing is a newer technique that may potentially be used for gene therapy. Instead of
adding new genetic material, genome editing introduces gene-editing tools that can change the
existing DNA in the cell. Genome editing technologies allow genetic material to be added,
removed, or altered at precise locations in the genome. CRISPR-Cas9 is a well-known type of
genome editing.
Is gene therapy safe?

- The first gene therapy trial was run more than thirty years ago.
- The earliest studies showed that gene therapy could have very serious health risks, such as
toxicity, inflammation, and cancer.
- Gene therapy techniques are relatively new, some risks may be unpredictable; however,
medical researchers, institutions, and regulatory agencies are working to ensure that gene
therapy research, clinical trials, and approved treatments are as safe as possible.
- Multiple levels of evaluation and oversight ensure that safety concerns are a top priority in the
planning and carrying out of gene therapy research.
Clinical trial Processes of Gene therapy

- Phase I studies determine if a treatment is safe for people and identify its side effects.
- Phase II studies determine if the treatment is effective, meaning whether it works.
- Phase III studies compare the new treatment to the current treatments available.
- Doctors want to know whether the new treatment works better or has fewer side effects than
the standard treatment. The FDA reviews the results of the clinical trial. If it determines that the
benefits of the new treatment outweigh the side effects, it approves the therapy, and doctors can
use it to treat a disorder.
What are the ethical issues surrounding gene therapy?

- The ethical questions surrounding gene therapy and genome editing include:
● How can “good” and “bad” uses of these technologies be distinguished?
● Who decides which traits are normal and which constitute a disability or disorder?
● Will the high costs of gene therapy make it available only to the wealthy?
● Could the widespread use of gene therapy make society less accepting of people who
are different?
● Should people be allowed to use gene therapy to enhance basic human traits such as
height, intelligence, or athletic ability?
Is gene therapy available to treat my disorder?
- Gene therapy is currently available primarily in a research setting.
- (FDA) has approved only a small number of gene therapy products for sale in the United
States.
- Gene therapies are available for conditions that include a rare eye disorder , a form of skin
cancer, and a genetic muscle condition . Other genetic therapies have been approved for blood
cell cancers such as lymphoma and multiple myeloma. Gene therapies to treat additional
conditions have been approved in other countries.

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Lesson 4

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Gene Therapy (A promising future to disease treatment)

Types of Gene Therapy

Germ Line Gene Therapy

What can it treat?

How does gene therapy work?

Is gene therapy safe?

Clinical trial Processes of Gene therapy

What are the ethical issues surrounding gene therapy?

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