The Aspects

of Gene
Therapy
Presented by:

Robert R. Maca
Christine Jay A. Relota
 Lesson Objectives
At the end of the lesson, students should be able to;
 Describe gene therapy and its forms.
 Know who is father of Genetics.
 Asses the issues potential benefits and detriments to
global health.
 Differentiate Somatic gene therapy to Stem Cell Gene
Therapy

2
Father of Genetics
Gregor Mendel was an Austrian monk who
discovered the basic principles of heredity through
experiments in his garden.

Mendel’s observations became the foundation of

modern genetics and the study of heredity, and he is
widely considered a pioneer in the field of genetics.
Gene Therapy
 Also known as “Gene Editing”
 A method that may treat or cure genetic-related
human illness.
 Based on correcting a disease at its root.
 It is used to remove “defective” genes and replace
them with a corrected copy.
Types of Gene Therapy
1.Somatic Gene Therapy
involves the manipulation of genes that will be
helpful to the patient but not inherited to the nest
generation.

2. Germ-Line Gene Therapy

 involves genetic modification of germ cells or
the origin cells that will pass the change on the
next generation.
2 Types of Somatic Gene Therapy
1. In Vivo Gene Therapy

 Gene is being transferred to a patient’s cells while in the

patient. However, a gene cannot be introduced directly into
patient’s cell. Instead, a carrier called a vector(virus) is
genetically engineered to transport the gene. Viruses are
frequently used as vectors for the reason that they are
effective at infecting cells and inserting the genes into the
cells’ DNA.
2 Types of Somatic Gene Therapy
1. Ex Vivo Gene Therapy

 Gene is being transferred into the cells in a laboratory and

then injected back into the patients via IV directly into a
specific tissue. Cells are being collected either by bone
marrow aspiration or by purifying blood drawn through a
central line in a process called apheresis.
 Basic Process of Gene Therapy
Replacement of mutated gene that causes disease
with a healthy copy of the gene

 Inactivation of a mutated gene that is functioning

improperly

 Introducing a new gene into the body to help fight a

disease
 STEM CELL GENE THERAPY

 are mother cells that have the potential to

become any type of cell in the body

 they have an ability to self-renew or multiply

 they can become cells of the blood, heart,

bones, skin, muscles and brains
 2 SOURCES OF STEM CELL

1.EMBRYONIC STEM CELLS- derived from a four-

or-five-day old human embryo, they usually extras
that are fertilized in a test tube of a women.

2.SOMATIC STEM CELLS- exist throughout the body

after embryonic development and found in the
different types of tissue.
Thank You
for
Listening!