Attendance

check!!!
MOTIVATION
AL ACTIVITY
 Guess the
gibberish!
Can you figure out the actual phrase from
the nonsense words?
Gibberish #1

thee wrath fee

Answer:

Therapy
Gibberish #2

dish older
Answer:

Disorder
Gibberish #3

beak tours
Answer:

Vectors
Gibberish #4

sum attic
Answer:

Somatic
Gibberish #5

this cyst
Answer:

Disease
Gibberish #6

choam moss rum

Answer:

Chromosome
 GENE
THERAPY
 Learning Outcomes
At the end of this module, the students
must have:
1. Described gene therapy and its various
forms;
2. Assessed the issues, potential benefits and
detriments to global health.
 Chapter outline
Genes and the Human
1 2 Genetic Disorders
Genome

Benefits and
3 Gene Therapy 4 Disadvantages of
Gene Therapy
Genes and
the human
genome
 Genome Chromosome DNA

 is a complete set of the  made up of two  This specific portion of the

genes in a cell or chromatids and each DNA performs specific
organism. chromatid contains function specifically in the
 Human genome resides in sequence of DNA production of hormone or
46 chromosomes that (deoxyribonucleic acid) enzymes
basically come in pair in  Not all the genes in the
which 23 are from the strand of DNA are
father and another 23 working at the same time.
from the mother.
 GeneTIC
DISORDERS
Genetic disorders are diseases caused by abnormalities in the
DNA sequence of an individual. These irregularities can happen in
just one gene or there is an addition or subtraction of chromosomes.
There are more than four thousand identified medical disorders that
are caused by defective genes. Although genes are responsible for
predisposition of these diseases the environment, diet and lifestyle
are also factors that trigger these diseases.
Single-Gene
Disorder

Chromosomal
Disorder

Multifactorial
Inheritance
 Single-Gene
Disorder
 are caused by mutations in specific genes.
 Single-gene disorders are classified into
autosomal single-gene disorder and X-
linked disorders.
 Autosomal gene disorder happens in
genes that can be found within the 22 pairs
of non-sex chromosomes while X-linked
disorder originated from the genes of sex
chromosomes.
 Chromosomal
Disorder
 Chromosomal may happen under certain
conditions.
 It might be an excess or deficiency of the whole
chromosome, chromosomal rearrangement, and
contiguous-gene syndrome.
 There are many structural abnormalities in the
chromosomes and some of it are related to
cardiovascular diseases that lead to congenital
heart disease.
 Chromosomal rearrangement happens if there
is breakage and reconstruction but in abnormal
form.
 Structural rearrangement, however, may lead
to loss or gain of genetic material but typically
gaining human chromosomes is less harmful than
the loss of genetic materials.
 Multifactorial
Inheritance
 Multifactorial inheritance is not caused by some

changes or abnormalities in a single gene or

chromosome.

 It is basically caused by the combination of

genetic changes that produces the disease.

 Multifactorial inheritance might happen 5-10% in

the first degree relatives with higher risk than that

of the 2nd degree relatives.

Gene therapy
 Gene Therapy
 a method of treating or preventing diseases by replacing,
repairing, or turning on or off genes of a patient’s cells.
 The process in gene therapy includes the use of vectors
and non-viral method to move a DNA or genes to one cell
or another.
Vectors:

RETROVIRUS
Vectors:
Vectors:
ADENOVIRUS
Vectors:
HERPES
SIMPLEX
VIRUS
Vectors:
Non-Viral method
 has certain advantages over vectors or viral methods since
it can be produced in large scale.
 Injection of naked DNA is the simplest method under non
viral-method.
 Cells, tissue and even organisms that undergone gene
therapy are considered transgenic or genetically modified.
 2 main types of gene therapy
Somatic Gene
1
Therapy

2
Reproductive Gene
Therapy
Somatic Gene Therapy
 It is basically the introduction of genes to cells in a target
organ to produce enzymes which are needed by the body
to function well.
 helps the organ to function well but it doesn’t alter the
genetic makeup of an organism.
 The changes in organisms caused by somatic gene therapy
are not transmitted to its offspring.
Reproductive Gene Therapy
 Also called “germ-line cell therapy” is basically the
introduction of corrective genes to sperm cells, egg cells or
even zygotes (4-day old).
 Its main goal or objective is to eliminate the abnormalities
that can possibly be transmitted to the offspring. It is the
alteration of genetic makeup of the offspring by targeting
the abnormal genes in the parent’s sex cells.
Take note!
GENE THERAPHY requires the identification of the correct genes
responsible in the progression of diseases. The most crucial part of gene
therapy is the delivery of the modified gene to the cell or target organ and
this should be done in accurate, controlled and effective way. It can also
be noted that modified genes are not always accepted by the cells for the
reason that our cells have its natural defense mechanism and kills foreign
bodies. Most failures in gene therapy are caused by the rejection of the
modified genes.
 Benefits &
Disadvantages of
gene therapy
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 multigene
disorder.
4. High cost.
5. Ethical issues.
Get a ¼ sheet of paper
and get ready for the
quiz. Erasure means
wrong. You’ll be given 1
minute to answer the
questions.
You have 5 minutes to review the lesson
Get a ¼ sheet of paper
and get ready for the
quiz. Erasure means
wrong. You’ll be given 1
minute to answer the
questions.
You have 5 minutes to review the lesson
Question 1: 1 minute

It is made up of two chromatids and each chromatid

contains sequence of DNA

A. Genes
B. Chromosome
C. Genome
D. Enzymes
Question 2: 1 minute

It is basically caused by the combination of genetic

changes that produces the disease.

A. Gene Therapy
B. Chromosomal Disorder
C. Single-gene Disorder
D. Multifactorial Inheritance
Question 3: 1 minute

The following choices are examples of Gene therapy

vectors, EXCEPT?

A. Retrovirus
B. Injection of naked DNA
C. Cis and Trans – acting element
D. Herpes simplex virus
Question 4: 1 minute

The following choices are examples of the benefits of

Gene therapy, EXCEPT?

A. It has a promising potential not only in the field of

medicine but in some related fields like agriculture.
B. It can replace defective cells.
C. Viral vectors could recover its ability to cause disease.
D. Has a promising potential not only in the field of
medicine but in some related fields like agriculture.
Question 5: 1 minute

________ are diseases caused by abnormalities in the

DNA sequence of an individual

A. Genetic Disorders
B. Genome
C. Viruses
D. Production of hormones
Question 6: 1 minute

It is a complete set of the genes in a cell or organism.

A. Virus
B. Genome
C. Cell
D. Enzymes
Question 7: 1 minute

The following choices are examples of the disadvantages

of Gene therapy, EXCEPT?
A. It can damage the gene pool resulting to multigene
disorder.
B. High cost.
C. Ethical issues.
D. It can replace defective cells.
Question 8: 1 minute

A method of treating or preventing diseases by replacing,

repairing, or turning on or off genes of a patient’s cells.

A. Gene Therapy
B. Cloning
C. Mutation
D. Clinical Trial
Question 9: 1 minute

How many chromosomes does a human have?

A. 23 chromosomes
B. 45 chromosomes
C. 46 chromosomes
D. 22 chromosomes
Question 10: 1 minute

It happens if there is breakage and reconstruction but in

abnormal form.

A. CAR T-cell therapy

B. Structural rearrangement
C. Combination therapy
D. Chromosomal rearrangement
DNA-TEST ICON-PAKET