• DNA

-DEOXYRIBONUCLEIC ACID is an organic chemical that

contains genetic information and instruction to protein
synthesis. It is found in most cells of every organisms. It
contains genetic heredity which is paasdown from parents to
offspring
• RNA
RIBONUCLEIC ACID is a nucleic acid present in all living cells
that has structural similarities to DNA however RNA is most
often single stranded. It is an important biological
macromolecule that functions to convert the genetic
information of DNA into proteins. It is essential in coding,
decoding, regulation and expression of genes.
 WHAT IS GENE THERAPY ?
• An experimental technique for correcting defective
genes that are responsible for disease development
• The most common form of gene therapy involves
inserting a normal genes to replace an abnormal gene.
• Other approaches used :
➢ Replacing a mutated gene that causes disease with a
healthy copy of the gene
➢ Inactivating or knocking out a mutated gene that is
functioning improperly
➢ Introducing a new gene into the body to help fight a
disease.
GENE THERAPY Stem cells. Stem cells are the cells from which all other
cells in your body are created. For gene therapy, stem
cells can be trained in a lab to become cells that can help
fight disease.

How does Gene Therapy work??

GENE THERAPY

• In 1972, Theodore Friedman and Richard Roblin proposed

that people with genetic disorders can be treated by
replacing defective DNA with good DNA.

• In 1985, Dr. W. French Anderson and Dr. Michael Blasse

worked together to show that cells of patients with
Adenosine deaminase (ADA) deficiency can be corrected in
tissue culture.
 HISTORY AND DEVELOPMENT OF GENE
THERAPY
• 1960: The concept of gene therapy was introduced
• 1970 Friedman and Roblin author of a paper in Science titled “Gene
therapy for the human genetic disease?” cite the first attempt to
perform gene therapy
• 1990
▪ The first approved gene therapy case at the National Institute of
Health, U.K. It was performed on a four year old girl named Ashanti
Dasilva. It was a treatment for a genetic defect that left her with an
immune system deficiency
▪ New gene therapy approach repairs errors in messenger RNA
derived from defective genes, This technique has the potential to
treat the blood disorder Thalassaemia, cystic fibrosis, and some
cancers
▪ Sickle cell disease is successfully treated in mice
GENE THERAPY
HISTORY AND DEVELOPMENT OF GENE
THERAPY
• 1992: Doctor Claudio Bordignon working at the Vita-Salute San Raffaelle
University, Milan, Italy, performed the first procedure of gene therapy
using hematopoietic stem cells as vectors to deliver genes intended to
correct hereditary diseases
• 1999: Death of Jesse Gelsinger in a Gene-therapy experiment resulted in a
significant setback to gene therapy research in the United States
• 2006: Scientists at the National Institute of Health (Bethesda, Maryland)
have successfully treated metastatic melanoma in two patients. This study
constitutes one of the first demonstrations that gene therapy can be
effective in treating cancer
• 2007-2011: Research is still ongoing and the number of diseases that has
been treated successfully by gene therapy increases.
✓ Retinal diseases
✓ Colour blindness
✓ Adrenoleukodystrophy
✓ 2011: Medical community accepted that it can cure HIV as in 2008. Gero
Hutler has cured a man from HIV using gene therapy
TYPES OF GENE THERAPY
SOMATIC GENE THERAPY:
• Transfer of a section of DNA to any cell of the body that doesn’t
produce sperm and eggs.
• Effects of gene therapy will not be passed onto the patient’s
children.

GERMLINE GENE THERAPY:

• Transfer of a section of DNA to cells that produce eggs or sperm.
• Effects of gene therapy will be passed onto the patient’s children
and subsequent generations.
 GERM LINE GENE THERAPY
• Result in permanent changes.
• Potential for offering a permanent therapeutic effect
for all who inherit the target gene.
• Possibility of eliminating some diseases from a
particular family
• Also raises controversy:
▪ Some people view this type of therapy as un natural
and liken it to “playing God”
▪ Others have concerns about the technical aspects.
 SOMATIC GENE THERAPHY
• Affects only the targeted cells in the patient, and
is not passed to future generations
• Short-lived because the cells of the most tissues
ultimately die and are replaced by new cells
• Transporting the gene to the target cells or tissue
is also problematic.
• Appropriate and acceptable for many disorders,
including cystic fibrosis, muscular dystrophy,
cancer, and certain infectious diseases.
SOMATIC GT and GERMLINE GT
Types of somatic gene therapy
• Cells are modified outside the
body and then transported back
Ex vivo in again
• Called ex-vivo because the cells
are treated outside the body

• Genes are changed in cells when

the cells are still in the body
In vivo • Called in-vivo because the genes
is transferred to cells inside the
patients body
In Vivo Gene Therapy
Ex Vivo Gene Therapy
 ADVANTAGES OF GENE THERAPY
• Give a chance of a normal life to baby born
with genetic disease
• Give hope of healthy life to cancer patient
• For certain diseases that do not have any cure
except gene therapy, it could save many lives
In 2016, the Committee for Medical Products for Human Use
(CHMP) of the European Medicines Agency (EMA) endorsed the
gene therapy treatment called Strimvelis that was approved by the
European Commission in June 2018.
DISAVANTAGES OF GENE THERAPY
• The genetic testing, screening and research in
finding the availability of certain genes is very
controversy.
• May increase rate of abortion if prenatal test
regarding baby with genetic disease is done
• The cost is very high and the patient might need
an insurance to cover the treatment
• Cosmetic industry may monopolized this gene
therapy if it is used in enhancing beauty and in
vanishing the aging effect rather than used for
treatment of a disease.
RISKS of Gene Therapy

• Unwanted immune system reaction

- your body’s immune system may see the newly
introduced viruses as intruders and attack them. This may cause
inflammation and, in severe cases, organ failure.

• Targeting the wrong cells

- because viruses can affect more than one type of cells, it’s
possible that the altered viruses may infect additional cells, not just
the targeted cells containing mutated genes. Healthy cells may be
damaged, causing other illness or diseases, such as cancer.
RISKS of Gene Therapy
• Infection caused by the virus
- it’s possible that once introduced into the body,
the viruses may recover their original ability to cause
disease.

• Possibility of causing a tumor

- if the new genes get inserted in the wrong spot in
your DNA, there is a chance that the insertion might lead
to tumor formation.
 ETHICAL QUESTIONS SURROUNDING
GENE THERAPY
• How can “good” and “bad” uses of gene therapy be
distinguished?
• Who decides which traits are normal and which
constitutes a disability or disorder?
• Will the therapy only benefit the wealthy due to its
high cost?
• Could the widespread use of gene therapy make the
society less accepting of people who are different?
• Should people allowed the use of gene therapy to
enhance basic human traits such as height,
intelligence, or athletic