The slide is about what is gene therapy and what are it's type. Role of gene therapy in conservation and treatment of various diseases.

1. Gene Therapy And Application
of Gene Cloning In Conservation
By – Akriti GAURAV

2. Contents
What is Gene Therapy And History
Types
Success Cases
Gene Cloning And Conservation

3. What is Gene Therapy ?
Gene therapy replaces a faulty gene or adds a new gene in an attempt to cure
disease or improve your body's ability to fight disease. Gene therapy holds
promise for treating a wide range of diseases, such as cancer, cystic fibrosis,
heart disease, diabetes, hemophilia and AIDS
The first attempt at modifying human DNA was performed in 1980 by Martin
Cline, but the first successful nuclear gene transfer in humans, approved by
the National Institutes of Health, was performed in May 1989.
The first therapeutic use of gene transfer as well as the first direct insertion
of human DNA into the nuclear genome was performed by French Anderson
in a trial starting in September 1990.
HISTORY

4. Types of Gene Therapy
SOMATIC GENE THERAPY
Somatic cell gene therapy
involves the placement of
a human gene into a
living person's somatic
cells—cells that do not
produce the eggs and
sperm that in turn
produce the next
generation.
GERM LINE GENE
THERAPY
Germline gene therapy is
the replacement of gene
in which
an offspring would inherit
a new trait. This type of
gene modification is still
in the early stages of
human medical
intervention.

5. EX VIVO GENE THERAPY
Isolate cells with
genetic defect
from a patient
Grow the cells in
culture
Introduce the
therapeutic genes
Select genetically
corrected cells
and grow
Transplant the
modified cells to
the patient
Example of Ex Vivo Gene Therapy
First Gene Therapy to correct deficiency of enzyme, (ADA)

6. Types of Somatic Cell Gene Therapy

7. IN VIVO GENE THERAPY
Direct delivery of therapeutic gene into target cell
Carried out by viral or non viral vector syestems
• Retroviruses
• Adeno viruses
• Adeno associated viruses
Viral
Vectors
• Gene Gun Method
• Electroporation
• Microinjections
Non Viral
Vectors

8. Germline therapy involves modifying the genes inside sperm or egg cells, so
that future children will have the modified genes. Although scientists
understand the science needed to perform this procedure, it is not currently
in practice due to ethical considerations of modifying human eggs and sperm.

9. Success cases of Gene Therapy
Immune
deficiencies
Severe
Combined
Immune
Deficiency
(SCID)
Adenosine
deaminase
(ADA)
deficiency
Hereditary
blindness

10. ADVANTAGES
• Gene therapy has
the potential to
eliminate and
prevent
hereditary
diseases such as
cystic
fibrosis,ADA- SCID
• It is a possible
cure for heart
diseases,AIDS and
Cancer
DISADVANTAGES
• Short lived
• Potential risk
• Viral toxicity

11. Applications of Gene Cloning in Conservation
In 2009 the Brazilian Agricultural Research Corp. (Embrapa) and the Brasilia Zoological Garden began scavenging and
freezing blood, sperm and umbilical cord cells from roadkill and other wild animals that had died, mostly in the Cerrado
savanna—an incredibly diverse collection of tropical forest and grassland ecosystems home to at least 10,000 plant
species and more than 800 species of birds and mammals, some of which live nowhere else in the world.
Specimens were collected from the bush dog, collared anteater, bison and gray brocket deer, among other species.
Successful cloning generally involves at least three essential components: DNA from the animal to
be cloned; a viable egg to receive that DNA; and a mother to gestate the resulting embryo.
In the early 1950s, at the Lankenau Hospital Research Institute in Philadelphia,
Robert Briggs and Thomas King successfully cloned 27 northern leopard frogs
through a process known as nuclear transfer.