ASPECTS OF GENE THERAPY

Lesson Objectives

• At the end of the lesson, the students should be able to:

• describe gene therapy and its various forms;

• assess the issue's potential benefits & detriments to
global health.
 Introduction

• Medical science has detected many human diseases

related to defective genes.

• These diseases cannot just be cured by taking readily

available medicines instead, gene therapy is a potential
method to either treat or cure genetic related human
illnesses.
 Introduction

• In 2015, team of
researchers from Harvard
Medical School & Boston
Children's Hospital
stated that “they were able
to restore the hearing of a
genetically deaf mice
using Gene Therapy.
 Introduction

• Boston Children's Hospital

(research team) also reported
that they were able to restore
a higher level of hearing in GE
mice up to 25 decibels.
• Anc80 was used to transfer
genes to inaccessible outer
hair cells when introduced into
cochlea( Fleisher 2017).
 History of Gene Therapy

• Started in 1971, when the

first rDNA were planned.
• Mechanism of Gene
therapy in the past:
insertion of foreign DNA into
a patient's tissue that hope
to succesfully eradicate the
targeted disease.
The Basic Process

• Replacement: mutated
gene to healthy copy
• Inactivation:mutant gene
functions improperly.
• Introduction: new gene to
help fight disease.
Recombinant DNA
 2 Types of Gene Therapy

• Somatic gene therapy

invoves the manipulation of
genes in cells that will be
helpfull to the patient but
not inherited to next
generation.
(Nimsergern,1988)
 Germ - line gene therapy

• Involves genetic
modification of germ cells
or the origin cells that will
pass the change on to
the next generation.
 The Concept of Stem Cells

• What is your understanding of stem

cells?

• What are the characteristics of stem cells

 Stem cell gene therapy

• Stem cells are the mother

cells that have the
potential to become any
type of cell in the body.
• It has the ability to self-
renew or multiply while
maintaining the potential to
develop into other types of
cells.
 Sources of Stem cells

• Embryonic stem cells

derive from a 4 or 5 day old
human embryo that is in the
blastocycst phase of
development.
- the embryos are usually extras
that have been created in IVF
where several eggs are fertilized
in a test tube then implanted into
a woman. (Crosta,2013)
 Somatic Stem Cel/ Adult Stem Cell

• Exist throughout the

body(after embryonic
development) and are
found inside different types
of tissues:
Brain,skeletal muscles,
blood vessels, bone
marrow, skin & liver.
Characteristics of Stem Cells
 The Bioethics of Gene Therapy
There are critical issues involved in gene therapy;
• How can good & bad uses of gene therapy be distiguished?
• Who decides which traits are normal & which constitute a
disability or disorder?
• Will the high cost of gene therapy make it available only to the
wealthy?
• Could the widespread use of gene therapy makes society less
accepting of people who are different?
• Should people be allowed to use gene therapy to enhance basic
human traits such as height, intellegence or athletic ability?