Topic 5: The Aspects of Gene Therapy

 Activity 1- Activite Prior Knowledge

Learning Activity: W-O-R-D-U-P!
Directions: Using the grid below, find words related to gene
therapy. You may use the statements that follow as your clues to
identify the words. Write your answer on the space provided
after each statement.

E Q Z X L E L L Q S T Y C W M
C G V C H T S I P K I K I U E
G O E B K H U N T I N G T O N
V L C R H O R U F H R A A N I
V W T Y M P I H C E T U M R O
J Q O K A L V F L I T E O R T
B L R L U L I R D W E T S E A
O E S M T T A N Q N T Y U O I
L S I C K L E C E L L A N E M
P S V E O U Q G A A E I O Z P

1. A disorder of the nervous system that usually occurs after the age of 45.
________________________
2. A disorder in the blood caused by an inherited abnormal hemoglobin (the oxygen-
carrying protein within the red blood cells. ________________________________
3. Are plasmid or viruses that are used to move recombinant DNA from one cell to
another. __________________________
4. It is the alteration of the nucleotide sequence of the genome of an organism, virus or
extrachromosomal DNA. ________________________________
5. A unit of DNA that usually located on a chromosome and that controls the development
of one or more traits. _____________________________________
6. Gene therapy that targets the reproductive cells meaning any changes made to the DNA
will be passed on the next generation. __________________________
7. It is when the DNS is transferred into body tissues. It specifically targets cells in the body
which are not passed on to the person’s children.
Gene therapy has become an increasingly important topic in science-related news. The aim of
gene therapy is the successful integration of correct, therapeutic DNA into the genomes of cells
to alter gene expression and, in turn, protein synthesis to correct genetic disease.

Two Types of Gene Therapy

Somatic gene therapy, which involves introducing a “good” gene into targeted cells to treat the
patient – but the patient’s future children because the genes do not get passes along to
offspring.
Germline gene therapy, involves the introduction of corrective genes into reproductive cells
(sperm and eggs) or zygotes, with the objective of creating a beneficial genetic change that is
transmitted to the offspring.

Vectors for Gene Delivery

Gene therapy vectors (Viral and non-viral)
Viruses, liposomes, and naked DNA  are some of the vehicles used to introduce transgene
into the host genome .
The vehicles used to introduce the transgene is known as vectors, various criteria to select
vector for gene therapy are discussed here. The utility of the vector depends on the factor
enlisted below,

 The size of the exogenous gene (transgene)

 The efficiency of the delivery
 It will induce the host immune response or not
 The stability and longevity of the transgene
 Level of expression of a transgene

“The transgene which is selected for the gene therapy should not induce an immune
response in the host, should have capacity to carry larger transgene, and must have a
high expression rate. “
Appropriate methods to deliver DNA used in gene therapy are vital, as the targeted tissues
must properly receive the appropriate genes. Gene therapy can be carried out using naked DNA
delivered directly Vectors for Gene Delivery.
The classification the gene therapy vectors are given into the figure below:

Adenovirus- are a group of common viruses that infect the lining of

your eyes, airways and lungs, intestines, urinary tract, and nervous
system. They’re common cause pf fever, coughs, sore
throats, diarrhea, and pink eye.

Structure of Adenovirus

Retrovirus is type of RNA virus that

inserts a copy of its genome into
the DNA of a host cell that it
invades, thus changing the genome
of that cell.
Adeno-associated virus (AAV) – is a non – enveloped virus that can be engineered to deliver
DNA to target cells, and has attracted a significant amount of attention in the field, especially in
clinical stage experimental therapeutic strategies.

Structure of Herpes Simplex

Herpes simplex type 1 cause’s sores around the mouth and lips
(sometimes called fever blisters or cold sores). HSV -1 can cause
genital herpes, but most cases of genital herpes are cause by
herpes type 2. In HSV-2, the infected person may have sores
around the genitals or rectum.

Naked DNA refers to DNA that is not associated with proteins, lipids, or any other molecule to
help protect it. Naked DNA is the result of release of genetic information into the surrounding
environmeent, such as from bursting cells.

DNA structure with magnified schematic of ribonucleotide.

The central molecule is a deoxyribose without the nitrogen base or phosphate group .
https://www.rfi.ac.uk/science-update/viewing-naked-dna/

https://bio.libretexts.org/Bookshelves/
Microbiology/Book
%3A_Microbiology_(Bruslind)/
17%3A_Microbial_Genetics
Gene Acquisition via Transformation.

Bioethical Issues of Gene Therapy

The first death association wwith gene therapy

occured on September 18, 1999, at the University
of Pennsylvania. Jesse Gelsinger was participating
in a clinical trial, a biomedical experiment for
evaluation of safety and eficiency of a therapy for
a disease. Gelsinger, who was 18 years old at the
time of the treatment, had the deficiency of
ornithine transcarboamylse , an important enzyme
in the metabolism of ammonia. Patients with this rare metabolic disorder must maintain a low-
protein diet and take a series of medicines to avoid ammonia poisoning in the blood stream.
The gene therapy Gelsinger took triggerd a chain reaction in his immune system, resulting in
hepatic respiratory failure, and consequently, his death four days after being treated.

Effects on the Environment

Although the positive impacts of gene therapy
could be enermous, there are many questions
raised that needs to be answered. New
organisms created by genetic engineering
could present an ecological problem. One
cannot predict the changes that a genetically
enginnered species would make on the
environment. The release of a new genetically
engineered species would also have the
possibility of causing an imbalance in the
ecology of a region just exotic species woul
do. An accident or an unknown result could cause several problems. An accident in engineering
the genetics of a virus or bacteria for example could result in a stronger type, which could cause
a serious epidemic when released. This could be fatal in human genetic engineering creating
problems ranging from minor medical problems, to death.

Effects on Human
Looking at the fact that gentic enginnerin employs
viral vector that carries functional gene inside the
human body; the repecussion are still unknown.
There are no clues as to where functional genes ae
being placed. They may even replace the mportant
genes, instead of mutated genes. Thus, this may
lead to another health condition or disease to
human. Also, as defective genes are replaced with
functional gene, then it is expected that there will
have identical genomes, the population as a whole will be susceptible to virus or any form of
diseases.
Antibiotic Resistance
Genetic engineering often uses
genes for antibiotic resistance
as “selectable markers.” Early
in the engineering prcess,
these markers identify clls that
have taken up foreign genes.
Although they have no further
use, the genes continue to be
expressed in plant tissues.
Most genetically engineered
plat foods carry fully
functioning antibiotic-
resistance genes. The presence
of antibiotic-resistance genes
in food could have lethal
effects. Therefore, eatin these
foods could reduce the
effectiveness of antibiotics to
fight disease when these
antibiotic are taken with
meals. More so, the resistance
genes could be transferred to
human or animal pathogens.,
making them impervious to
antibiotics. If transfer were t occur, it could
aggravate the already serous health problem of
antibiotic resistant disease organisms.
Ethical and Social Issues
“Playing God” has become a strong argument
againts genetic engineering. Several issues have
also been raised as regards the acceptance of this
technology. These concerns range from ethical
isssues to lack of knowledge on the effects genetic
enginnering may have. One major concern is that
once an alterd gene is placed in an organism, the
process cannot be reversed. The production of
medicines through the use of genetically alterd
organisms might develop extremely infectious form
that could cause worldwide epidemics.
Activity 2: Make a definition and ideas of the following diseases:
 HIV
 Muscular dystrophy
 Alzheimer’s disease
 Cancer
 Tay-sach’s disease
 Cystic fibrosis
 Rheumatoid arthritis
 Ulcer
  Sickle cell anemia
 Hemophilia
 Coronary heart disease
 Bone fracture
 Huntington’s disease

Activity 3: Vector Voyage

Directions: Using the library or online resources, complete the table below. ( you may check in
Google)
Herpes
Retrovirus Adenovirus Simplex Virus Naked DNA
How the vector
carries genetic
material

Maximum length
of DNA that can
be inserted in the
vector

Advantages

Disadvantages

Activity 4: GENETIC DISORDER

Direction: Complete the table below. List down ten common genetic disorders. You may check
Google.
Genetic Disorder Gene/Defect Clinical Features
Guide Questions:
1. What do you know about gene therapy?
2. Do you think that FDA should allow the use of gene therapy in the Philippines? If yes
why? If no, why?
3. Do you think the government should allow and fun scientific researches on human
cloning? Why?

 Activity 5- Picture interpretation

What does the picture show?