BASIC PROCESS OF GENE THERAPY

Hercie’s part:

How does Gene Therapy work?

- A healthy gene is inserted into a carrier, called vector, and transferred to the
affected cells, either inside or outside the body.

Steps of gene therapy

The three steps of gene therapy are as follows:

1. Gene identification: This involves the identification of the gene of interest.

2. Gene duplication: This involves duplicating the gene of interest to be used for
therapy.
3. Gene insertion: The duplicated gene of interest is inserted into the recipient.

Hercie’s script:
- The basic process of gene therapy, how does gene therapy work? A healthy gene is
inserted into a carrier, called vector, and transferred to the affected cells, either inside
or outside the body. Dito sa gene therapy, ang mga doctors ay nagdedeliver ng isang
healthy copy ng isang gene sa mga cells sa loob or labas ng katawan ng isang tao and
then yung isang malusog na gene na yon ay pwede or maaari niyang palitan yung
nadamage or mutated na gene sa katawan ng isang tao. Yung tinatawag na vector ay
isang carriers or nagdadala ng mga healthy genes into cells.
- The Figure shows the transfer of therapeutic genes to the targeted cells is described
on the reverse side.
- Next here are the three steps of gene therapy, first Gene identification,this involves the
identification of the gene of interest. Dito nakadepende yung paghahanap ng isang
 locus gene para mapinpoint ang location nito. Second is Gene duplication, this involves
duplicating the gene of interest to be used for therapy. Ito yung process na kinokopya
yung isang area ng DNA coding para sa isang gene. Pero ang gene duplication ay
maaaring mag-occur as the result of an error in recombination or through a
retrotransposition event. Duplicate genes are often immune to the selective pressure
under which genes normally exist and the last step is Gene insertion, the duplicated
gene of interest is inserted into the recipient. Gene insertion is the addition of one or
more genes into a DNA sequence, ito ay isang pamamaraan na traditional na
ginagawa gamit ang plasmid DNA o pagsasama ng mga viral vector.
- The next slide will be discussed by Miss Hillary.

Hillary’s part:
There are several approaches to a gene therapy. These are the following therapy
(Pawilen, 2018):

Gene therapy, a groundbreaking medical field, uses several techniques to combat genetic
disorders (Pawilen, 2018):

1. Gene Replacement: This method replaces a disease-causing mutated gene with a healthy
copy, restoring normal function.

2. Gene Inactivation: This approach involves deactivating a malfunctioning gene, preventing

it from causing further harm.

3. New Gene Introduction: This strategy introduces a new gene into the body, providing a
new tool to fight disease.
However, the process of inserting a therapeutic gene into a human cell is not straightforward.
It requires a carrier, or vector. The most common vectors today are viruses that have been
genetically altered to carry normal human DNA. These viruses have a natural ability to
encapsulate and transport their genes into human cells, a trait that scientists have
harnessed for therapeutic use.

Gene therapy is a promising field with the potential to revolutionize the treatment of genetic
disorders. Despite the challenges, the use of vectors for gene delivery presents a viable
strategy for disease treatment and prevention.