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Gene Therapy

Gene therapy is an experimental technique that treats or prevents disease by adding new copies of healthy genes or replacing defective genes. It works by altering the genetic code to recover the functions of critical proteins. There are two main approaches: somatic cell gene therapy targets non-reproductive cells, while germline cell therapy aims to alter heritable genes but poses greater safety risks. Diseases treated with gene therapy include genetic disorders caused by single-gene defects like cystic fibrosis and sickle cell anemia, as well as some cancers and infectious diseases. However, gene therapy also carries risks such as unwanted immune reactions, targeting the wrong cells, viral infection, and potential tumor formation.

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Jienesa Canoy
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120 views

Gene Therapy

Gene therapy is an experimental technique that treats or prevents disease by adding new copies of healthy genes or replacing defective genes. It works by altering the genetic code to recover the functions of critical proteins. There are two main approaches: somatic cell gene therapy targets non-reproductive cells, while germline cell therapy aims to alter heritable genes but poses greater safety risks. Diseases treated with gene therapy include genetic disorders caused by single-gene defects like cystic fibrosis and sickle cell anemia, as well as some cancers and infectious diseases. However, gene therapy also carries risks such as unwanted immune reactions, targeting the wrong cells, viral infection, and potential tumor formation.

Uploaded by

Jienesa Canoy
Copyright
© © All Rights Reserved
Available Formats
Download as DOCX, PDF, TXT or read online on Scribd
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Answer the following questions?

1.What is gene therapy?


Gene therapy is an experimental technique that uses genes to treat or prevent disease.
Often, gene therapy works by adding new copies of a gene that is broken or by replacing a
defective or missing gene in a patient’s cells with a healthy version of that gene. Both inherited
genetic diseases (e.g., hemophilia and sickle cell disease) and acquired disorders (e.g., leukemia)
have been treated with gene therapy. Gene therapy is a direct way to treat genetic conditions as
well as other conditions. 

2. How does gene therapy works? Discuss how it works.


Gene therapy works by altering the genetic code to recover the functions of critical
proteins. Proteins are the workhorses of the cell and the structural basis of the body’s tissues. The
instructions for making proteins are carried in a person’s genetic code, and variants (or
mutations) in this code can impact the production or function of proteins that may be critical to
how the body works. Fixing or compensating for disease-causing genetic changes may recover
the role of these important proteins and allow the body to function as expected.
Gene therapy can compensate for genetic alterations in a couple of 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.

3. Are there different approaches in gene therapy? Please specify.


There are two approaches to achieve gene therapy.
a. Somatic Cell Gene Therapy. The non-reproductive (non-sex) cells of an organism are
referred to as somatic cells. These are the cells of an organism other than sperm or eggs
cells, e.g., bone marrow cells, blood cells, skin cells, intestinal cells. At present, all the
research on gene therapy is directed to correct the genetic defects in somatic cells. In
essence, somatic cell gene therapy involves the insertion of a fully functional and
expressible gene into a target somatic cell to correct a genetic disease permanently.
b. Germ Cell Gene Therapy. The reproductive (sex) cells of an organism constitute germ
cell line. Gene therapy involving the introduction of DNA into germ cells is passed on to
the successive generations. For safety, ethical and technical reasons, germ cell gene
therapy is not being attempted at present.
The genetic alterations in somatic cells are not carried to the next generations. Therefore,
somatic cell gene therapy is preferred and extensively studied with an ultimate objective of
correcting human diseases. Development of gene therapy in humans for any specific disease
involves the following steps. In fact, this is a general format for introducing any therapeutic
agent for human use.
i. In vitro experiments and research on laboratory animals (pre-clinical trials).
ii. Phase I trials with a small number (5-10) of human subjects to test the safety of the product.
iii. Phase II trials with more human subjects to assess whether the product is helpful.
iv. Phase III trials in large human samples for a final and comprehensive analysis of the safety
and efficacy of the product.

4. Which diseases are treated by gene therapy? List them down.


Gene Therapy was initially meant to introduce genes straight into human cells, focusing
on diseases caused by single-gene defects, such as cystic fibrosis, hemophilia, muscular
dystrophy (see video 2) and sickle cell anemia (see also Wiley database on indications addressed
by gene therapy clinical trials). Three types of diseases for gene therapy can be distinguished:
i. Monogenic disorders, single locus (gene) is defective and responsible for the disease, 100%
heritable. Examples: Sickle cell anemia, Severe Combined Immunodeficiency (ADA-SCID / X-
SCID), Cystic fibrosis, Hemophilia, Duchenne muscular dystrophy, Huntington’s disease,
Parkinson’s, Hypercholesterolemia, Alpha-1 antitrypsin, Chronic granulomatous disease,
Fanconi Anemia and Gaucher Disease.
ii. Polygenic disorders, multiple genes involved, disease may be dependent on environmental
factors and lifestyle. Examples: Heart disease, Cancer, Diabetes, Schizophrenia and Alzheimer’s
disease.
iii. Infectious diseases, such as HIV.

5. Are there risks associated with gene therapy? State if there are.
Yes, Gene therapy has some potential risks. A gene can't easily be inserted directly into
your cells. Rather, it usually has to be delivered using a carrier, called a vector. The most
common gene therapy vectors are viruses because they can recognize certain cells and carry
genetic material into the cells' genes. Researchers remove the original disease-causing genes
from the viruses, replacing them with the genes needed to stop disease.

This technique presents the following risks:

● Unwanted immune system reaction. Your body's immune system may see the newly
introduced viruses as intruders and attack them. This may cause inflammation and, in severe
cases, organ failure.

● Targeting the wrong cells. Because viruses can affect more than one type of cells, it's
possible that the altered viruses may infect additional cells — not just the targeted cells
containing mutated genes. If this happens, healthy cells may be damaged, causing other
illnesses or diseases, such as cancer.

● Infection caused by the virus. It's possible that once introduced into the body, the viruses may
recover their original ability to cause disease.

● Possibility of causing a tumor. If the new genes get inserted in the wrong spot in your DNA,
there is a chance that the insertion might lead to tumor formation.

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