Activity 1

Directions: Using the library or online resources, complete the table below.

Retrovirus Adenovirus Herpes Simplex Naked DNA

Virus
How the In the form of RNA double stranded double stranded Plasmid
vector DNA DNA
carries genetic
material
Maximum 8, 000 base pairs 7, 500 base pairs 20, 000 base pairs no maximum
length length
of DNA that
can
be inserted in
the
vector
Advantages can target specific can infect both will not disrupt no immune
cells by engineering dividing and non- function of other response
proteins to its dividing cells genes in cell isn't toxic
surface possible to target stays in cell for a better for ex
retrovirus contains specific cell types long time vivo
enzymes that immune response immune response
converts RNA can be combatted can be combatted
material to DNA by removing by removing
material proteins from the proteins from the
immune response surface surface
can be combatted
by removing
proteins from the
surface
Disadvantages can only target has possibility of can cause immune integration
dividing cells causing an immune response in patient effectiveness is
can affect cell's response very low
ability to divide will not integrate enters cells less
promoting tumor into host cell's effectively
growth genome, will be doesn't target
can cause immune discarded after a specific cells
response week or two

Activity 2

Direction: Complete the table below. List down ten common genetic disorders.

Genetic Gene/Defect Clinical Features

Disorder
1. Down About 95 percent of the time, Short neck, with excess skin at the back of the
Syndrome Down syndrome is caused neck. Flattened facial profile and nose. Small
 by trisomy 21 — the person has
three copies of chromosome 21,
instead of the usual two copies,
in all cells. This is caused by
abnormal cell division during the
development of the sperm cell or
the egg cell. Mosaic Down
syndrome.
2. Edwards Edwards syndrome is a genetic
Syndrome condition in babies that causes
severe disability. It is caused
by an extra copy of chromosome
18 and babies born with the
condition usually do not survive
for much longer than a week.
3. Patau Patau's syndrome is a serious
Syndrome rare genetic disorder caused by
having an additional copy of
chromosome 13 in some or all of
the body's cells. It's also called
trisomy 13. Each cell normally
contains 23 pairs of
chromosomes, which carry the
genes you inherit from your
parents.
4. Turner Turner syndrome, a condition
Syndrome that affects only females, results
when one of the X chromosomes
(sex chromosomes) is missing or
partially missing. Turner
syndrome can cause a variety of
medical and developmental
problems, including short height,
failure of the ovaries to develop
and heart defects.
5. klinefelter's Klinefelter syndrome can be
syndrome caused by: One extra copy of the
X chromosome in each cell (XXY),
the most common cause. An
extra X chromosome in some of
the cells (mosaic Klinefelter
syndrome), with fewer
symptoms. More than one extra
copy of the X chromosome,
which is rare and results in a
severe form.
6. Fragile Site fragile site, folic acid type, rare,
head, ears, and mouth. Upward slanting eyes,
often with a skin fold that comes out from the
upper eyelid and covers the inner corner of the
eye.
Babies born with the Edwards syndrome often
have petite heads, clinched fists and curved
feet, and may have kidney, heart and skeletal
issues. A lot of babies with Edwards syndrome
die before birth, and of those born alive have
90% chances of death within the very first
year.
Many of the clinical features widely vary;
however, severe mental deficiency is a
consistent feature in children born with Patau
syndrome. Holoprosencephaly, polydactyly,
flexion of the fingers, rocker-bottom feet,
facial clefting, neural tube defects, and heart
defects are also frequent clinical features
Wide or web like neck.
Low-set ears.
Broad chest with widely spaced nipples.
High, narrow roof of the mouth (palate)
Arms that turn outward at the elbows.
Fingernails and toenails that are narrow and
turned upward.
Taller than average stature. Longer legs,
shorter torso and broader hips compared with
other boys. Absent, delayed or incomplete
puberty. After puberty, less muscle and less
facial and body hair compared with other
teens.
Fragile X syndrome is characterized by
 Fra(X)(Q27.3)
marker X syndrome
Martin-Bell syndrome
7. waardenburg Waardenburg syndrome types I
syndrome and III are caused by mutations in
the PAX3 gene. Mutations in the
MITF or SNAI2 gene can cause
Waardenburg syndrome type II.
Mutations in the SOX10, EDN3,
or EDNRB gene can cause
Waardenburg syndrome type IV.
8. lesch nyhan The only gene known to be
syndrome associated with Lesch-Nyhan
syndrome is located on the X
chromosome and called HPRT1.
Abnormalities (mutations) in the
HPRT1 gene result in the absence
or deficiency of the enzyme
hypoxanthine-guanine
phosphoribosyl transferase
(HPRT) and the abnormal
accumulation of uric acid in the
blood.
9. cri du chat Cri-du-chat (cat's cry) syndrome,
also known as 5p- (5p minus)
syndrome, is a chromosomal
condition that results when a
piece of chromosome 5 is
missing. Infants with this
condition often have a high-
pitched cry that sounds like that
of a cat. The disorder is
characterized by intellectual
disability and delayed
development, small head size
(microcephaly), low birth weight,
moderate intellectual disability in affected
males and mild intellectual disability in
affected females. Distinctive physical features
are sometimes present in affected males
including a large head, long face, prominent
forehead and chin, protruding ears, loose
joints and large testes, but these features
develop over time and may not be obvious
until puberty. Motor and language delays are
usually present but also become more
apparent over time. Behavioral abnormalities
including autistic behaviors are common.
Cleft lip (rare)
Constipation.
Deafness (more common in type II disease)
Extremely pale blue eyes or eye colors that
don't match (heterochromia)
Pale color skin, hair, and eyes (partial albinism)
Difficulty completely straightening joints.
Possible slight decrease in intellectual
function.
The symptoms of Lesch-Nyhan syndrome
include impaired kidney function, acute gouty
arthritis, and self-mutilating behaviors such as
lip and finger biting and/or head banging.
Additional symptoms include involuntary
muscle movements, and neurological
impairment.
The clinical symptoms of cri du chat syndrome
usually include a high-pitched cat-like cry,
mental retardation, delayed development,
distinctive facial features, small head size
(microcephaly), widely-spaced eyes
(hypertelorism), low birth weight and weak
muscle tone (hypotonia) in infancy.
 and weak muscle tone
(hypotonia) in infancy. Affected
individuals also have distinctive
facial features, including widely
set eyes (hypertelorism), low-set
ears, a small jaw, and a rounded
face. Some children with cri-du-
chat syndrome are born with a
heart defect.
10.Rett In 1999, NICHD-supported Slowed growth. Brain growth slows after
Syndrome scientists discovered that most birth. ...
classic Rett syndrome cases are Loss of normal movement and coordination. ...
caused by a mutation within the Loss of communication abilities. ...
Methylcytosine-binding protein 2 Abnormal hand movements. ...
(MECP2) gene. The MECP2 gene Unusual eye movements. ...
is located on the X chromosome. Breathing problems. ...
Between 90% and 95% of girls Irritability and crying. ...
with Rett syndrome have a Other abnormal behaviors.
mutation in the MECP2 gene.

Direction: Answer the following questions

1. Is gene therapy safe to cure genetic disorder?

Gene therapy is being researched to see if it can be used to treat disease. Gene therapy is now being
studied to see if it is safe, and future studies will see if it is an effective therapeutic option. Several
studies have indicated that this technique can have major health consequences, including toxicity,
inflammation, and cancer. Because the procedures are new, some hazards are unknown; nonetheless,
medical researchers, institutions, and regulatory bodies are attempting to make gene therapy research
as safe as possible.

2. If you are the patient suffering from a genetic disease are you willing to undergo gene therapy?

Why?

Yes, because Gene therapy is a type of treatment in which genes are used to treat or prevent disease.
Instead of treating symptoms and diseases with standard drugs and surgery, it offers the option of
altering genes inside or outside a patient's cells in an effort to treat and eventually eliminate the
condition. Gene therapy, on the other hand, is used to fix faulty genes in order to cure an illness or help
the body fight disease more effectively. It is the insertion, removal, or modification of genetic material—
specifically, DNA or RNA—into a patient's cells with the goal of restoring normal function or treating a
specific condition.

3. Do you think that FDA should allow the use of gene therapy in the Philippines? If yes why? If no,

why?
Yes, because the FDA is responsible for safeguarding public health by ensuring the safety, efficacy, and
security of human and veterinary pharmaceuticals, biological products, and medical devices, as well as
the safety of our country's food supply, cosmetics, and radiation-emitting goods.

4. Do you think the government should allow and fun scientific researches on human cloning? Why?

No, because some scientific experiments are potentially risky and may cause harm to others. For nearly
seven years, it has been able to clone human embryos. No one has tried because it is illegal, and it
would have been done in secret owing to the anger.