Gene Therapy Science Technology Amp Society Compress

GENE THERAPY
GENE
* THE BASIC UNIT OF HEREDITY
PASSED FROM PARENT TO CHILD.
* MADE UP OF SEQUENCES OF DNA
1990 - The first approved gene therapy case
at the National Institute of Health, UK. It was
performed on a four year old named Ashanti
DaSilva. It was a treatment for a genetic
defect that left her with an immune system
deficiency.
WHAT IS GENE THERAPY?
- A type of experimental
treatment in which foreign
genetic material ( DNA) is
inserted into a person’s cells to
prevent or fight disease. Gene
therapy is being studied in the
treatment of certain types of
Basic Process of Gene Therapy
-Replacing a mutated gene that
Several
causes disease with a healthy copy of
approaches to
the gene
gene therapy
are being - Inactivating, or “knocking out,”
tested, a mutated gene that is functioning
including: improperly
- Introducing a new gene into the body
to help fight a disease
In general, a gene cannot be
directly inserted into a
person’s cell. It must be
delivered to the cell using a
carrier, or vector.
TYPES
OF
GENE
THERAP
Y
Somatic Gene Therapy
- Obtaining blood cells with a genetic
disease then introduce a normal gene into
the defective cell
- Has to be done several times because the
effects does not last long, can't pass to the
next generation.
This therapy aims to reduce symptoms but will not be
passed to the patient’s children.
Germline Gene Therapy
- Involves genetic modification of the
gametes (reproductive cells, the egg cells and
the sperm cells) that will pass to the next
generation
- Can be done once because it is
permanent. This change would prevent the disorder
from being passed to future generations.
Vectors - vehicles that delivers the gene
to the cells
Viruses - have the natural ability to

deliver genetic materials into cells.
Retrovirus - inserts its genetic code

directly into the chromosomes of the host
cell
Adenovirus - introduce the
deoxyribonucleic acid (DNA) they carry to
the nucleus but not into the chromosomes
Fatty Liposomes and Directly injecting to

the skin cells or tumor cells - not as
effective as the viruses used.
Ex Vivo - outside the living, cells
are modified outside the body and
then transplanted back again.
In Vivo - inside the living, vector
can be injected or given
intravenously (IV) directly to the
specific tissue in the body
Stem Cell Gene Theraphy
Stem Cell – are the mother cells that
have the potential to become any type of
cell in the body. They have the ability to self-
renew or multiply.
Ex vivo, isolating the stem cells and
genetically correcting them, then transplanting
it back in the body with the goal of producing
genetically corrected protein in vivo
Embryonic Cells - cells that are self-replicating,
derived from human embryos or human petal
tissue and able to differentiate into 3 primary
germ cell layers, and into any type of cell in the
body
Somatic Cells - Any cell of the body
except sex cells. Can differentiate only into
different cell types present in the tissue of
their origin
ADVANTAGES & DISADVANTAGES
List of the Advantages of
Gene Therapy
1. It offers the possibility of a
positive medical outcome.
2. We can treat disease or
illness in meaningful ways.
3. It could improve a person’s
life in other ways
4. We already know that it can be a
successful treatment option.
5.There are out-of-body treatment

options available.
6.Gene therapy could be useful in

the treatment of multiple diseases.
7.The medical impact of gene therapy
can create permanent results.
8.New treatment options are coming out

all of the time.
9.It can work in combination with

cell therapy techniques.
List of the Disadvantages of Gene
Therapy
1.There could be unwanted
immune system reactions.
2.Current gene therapy methods
can sometimes target the wrong
cells.
3.The delivery viruses might
recover their ability to create disease.
4.Gene therapies could cause a
potential tumor.
5.There is no guarantee that the

gene therapy will work .
6.Some patients might

experience incompatibility issues.
7.Humans could develop a resistance
to the gene therapy.
8.The cost of gene therapy is prohibitive

to a number of families.
9. Ethical concerns about gene therapy

exist
10. It typically works best in single-gene
mutations.
11. There are funding challenges

to consider in this category.

Gene Therapy Science Technology Amp Society Compress

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GENE THERAPY

Viruses - have the natural ability to

Retrovirus - inserts its genetic code

Fatty Liposomes and Directly injecting to

5.There are out-of-body treatment

6.Gene therapy could be useful in

8.New treatment options are coming out

9.It can work in combination with

5.There is no guarantee that the

6.Some patients might

8.The cost of gene therapy is prohibitive

9. Ethical concerns about gene therapy

11. There are funding challenges

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