CHAPTER IX: THE ASPECTS

OF GENE THERAPY

THINK ABOUT THESE QUESTIONS:

Directions: Answer the questions and activities
.
based on the topic
discussed and understanding.

1. What is a Gene Therapy?

- Gene therapy is an approach that seeks the correct treatment of
alterations within the DNA itself. Instead of relying on medications or
operations, doctors can provide treatments by modifying a person's
genetics through gene therapy techniques.

2. What is a DNA like?

- Deoxyribonucleic acid, or DNA, is a lengthy molecule containing an
organism's organism's unique genetic code. It serves as a blueprint for the
construction of proteins present in the body. DNA contains four
fundamental bases, which are adenine (A), cytosine (C), guanine (G), and
thymine (T). DNA is uniquely structured as a two stranded molecule that
takes on the shape of a twisted ladder, or a double helix.

3. Would you subject yourself for gene therapy without its 100%
assurance of effectiveness or future negative side effects?
- I usually decide base on empirical evidences and doesn’t take risk unless
there’s a certainty in it and that there is no 100% assurance about the
effectiveness or future negative effects, I would not subject myself for gene
therapy yet for now. I would still weigh things in the future for a better
judgment whether I would take it or not.
 CHAPTER IX: THE ASPECTS
OF GENE THERAPY
4. Should gene therapy be limited to medical concerns only or could it
be used for aesthetic purposes?
- I strongly think that there is more to gene therapy than simply a medical
.
issue because this advancement in science can also be utilized for beauty
purposes. If there is a budget in spending and a willingness to undergo the
process, all we need to do is assist them in understanding the reasoning
behind opting for such a step and allow them to make their own choices.
Regardless, this process still needs to observe the basic principles that
govern the practice of medicine.

5. What do you know about a mutated gene?

- A gene mutation is a change in one or more genes. Some mutations can
lead to genetic disorders or illnesses. These mutations could happen
because of: 1) a change in one or more nucleotides of DNA, 2) a change in
many genes, 3) loss of one or more genes and 4) rearrangement of genes or
whole chromosomes.

6. It involves the genetic modification of germ cells. It is called?

- This process in called germline gene therapy which would change the
genetic makeup of the egg or sperm of an individual and would be carried
on to future generations.

7. From where did a Genes or DNA come from?

- Genes are the basic unit of living organisms’ inheritance. Gene comes
from my parents. We can inherit the probability of obtaining specific
diseases and conditions from our physical properties and parents. The gene
comprises data necessary to create and maintain cells and provide genetic
information to offspring. Each cell contains two chromosomal statements.
Male sperm and female eggs wear two sets of single sets, including 22
automatic bones and X- or Y sex chromosomes. Women inherit X
chromosomes from their parents, but a man inherited her mother's X
chromosome and Y chromosome from her father.
 CHAPTER IX: THE ASPECTS
OF GENE THERAPY

8. How can we use Gene therapy?

- Gene therapy can be used to modify cells inside or outside the body.
When done in the body, doctors inject the .gene-carrying vector directly
into the defective part of the body. Gene therapy, which is used to modify
cells outside the body, takes blood, bone marrow, or other tissue from the
patient and isolates specific cell types in the laboratory. Introduce a vector
containing the gene of interest into these cells. The cells are left in the
laboratory to proliferate, and then infused into the patient, where they
continue to proliferate and ultimately produce the
desired effect.

9. What kind of issue that a gene can be involved?

- A genetic disorder happens when a gene (or genes) has a problem with its
code, and this causes a health problem. Sometimes a genetic disorder
happens when a child inherits it from one or both parents. Other times, it
happens only in the child (and the parents do not have the genetic
disorder). Different things can cause a genetic disorder, such as 1) a change
(mutation) in one gene on a chromosome, 2) a missing part of a
chromosome (called a deletion), 3) genes shifting from one chromosome to
another (called a translocation), 4) an extra or missing chromosome and 5)
too few or too many sex chromosomes.

10. Is it safe to use a Gene Therapy?

- The first gene therapy research was conducted over 30 years ago. Early
studies have shown that gene therapy can pose very serious health risks
such as toxicity, inflammation, and cancer. Since then, researchers have
studied the mechanism and developed improved technologies that are less
likely to cause dangerous immune responses and cancer. Because gene
therapy technology is relatively new, some risks may be unpredictable.
However, medical researchers, institutions, and regulators are working to
ensure that gene therapy research, clinical trials, and approved therapies
are as safe as possible.
Flowchart. Make a flow chart of the basic process of gene therapy. Explain
each part of the process. You may use references for your guide.

Identify the Defective Gene

Scientists determine which gene mutation is causing the disease.
Genetic testing and sequencing are used to analyze the patient’s
DNA.

Design the Corrective Gene

A healthy version of the gene is created or extracted.
The therapeutic gene is designed to replace, repair, or
supplement the defective gene.

Select the Gene Delivery Method

A vector (carrier) is chosen to deliver the gene into the
patient’s cells.Common vectors include viral vectors
(e.g., adenovirus, lentivirus) or non-viral methods (e.g.,
lipid nanoparticles, direct injection).

Insert the Gene into Target Cells

The therapeutic gene is introduced into the patient's
cells either inside the body (in vivo) or outside the body
(ex vivo). In ex vivo therapy, patient cells are modified in
a lab before being reintroduced.

Monitor & Evaluate Treatment

The patient's response to therapy is tracked.
Scientists check for gene expression, effectiveness, and possible
side effects (such as immune responses).
 Concept mapping. Using a Venn diagram,
differentiate the two forms of gene therapy

SOMATIC GENE THERAPY GERMLINE THERAPY

Both involve
1. Involves obtaining blood altering specific 1.Takes place in the
cells from a person with a genes to correct reproductive cells. It
genetic disease and then mutations or
improve function. involves the genetic
introducing a normal gene
into the defective cell. modification of germ cells
Both aim to treat
or prevent that will pass the change
2. Does not prevent the disease genetic
disorders.
on to the next generation.
from occurring in the next
generation. Both may use
gene editing 2) Only has to be done one
tools like CRISPR-
3. Has to be done several times Cas9, viral time
over the coarse of the patient's vectors, or other to be permanent.
gene delivery
life because the effects do not methods.
last very long