Gene Therapy is the future of our health care & medicine. It is getting popular day by day.

1. An Insight into Human Gene Therapy: A Review
Roll No: 18
Reg. No: 225607
Session: 2014-15
Batch: 16th
Submission Date: 15.07.2018
Department of Pharmacy
DHAKA INTERNATIONAL UNIVERSITY

2. CONTENTS
Aim of the study
Definition of gene therapy
Types of gene therapy
Vectors used in gene therapy
Uses
Result and Discussion
Conclusion
References.

3. AIM OF THE STUDY
Gene Therapy is the best way to treat complex,
genetic diseases. The main aim of this study is
to assimilate proper knowledge about gene
therapy and analyze its future as a convenient
therapy to treat life threatening complications.

4. DEFINITION
The introduction of therapeutic genes into
the cells of the patient is called gene
therapy .

5. TYPES OF GENE THERAPY
Depending upon the target cell type gene
therapy is two types:
1. Germ-line gene therapy
2. Somatic gene therapy .
Depending upon the type of administration
gene therapy is two types:
1. Ex-vivo gene therapy
2. In-vivo gene therapy.

6. VECTORS USED IN GENE THERAPY
There are three common viral vectors are
used for gene therapy :
1.Retrovirus
2.Adenovirus
3.Adeno-associated virus.

7. Uses of Gene Therapy
 Cancer
 Allergy and Asthma
 Blindness
 Wilson’s disease, a rare pathology
caused by the lack or malfunction of a
gene
 Severe Combined Immune Deficiency
(SCID).

8. 70%
14%
10%
6%
Cancer Monogenic Diseases Infectious Diseases Other diseases
Figure: Proportion of protocol for human gene therapy
trials relating to various types of diseases.

9. 4%
65%
24%
6%
Multi-country
America
Europe
Asia
Australia
Africa

10. 5%
63%
9%
4%
3%
2%
2%
7%
Multi-country
USA
UK
Germany
China
France
Switzerland
Japan
Netherlands
Australia
Spain
Other countries
Figure: Geographical distribution of gene therapy
(country wise).

11. 65%
11%
7%
7%
2%
2%2%2% Cancer Diseases
Monogenic Diseases
Infectious Diseases
Cardiovascular Diseases
Neurological Diseases
Ocular Diseases
Inflammatory Diseases
Other diseases
Gene Marking
Healthy volunteers
Figure: Indication addressed by gene therapy.

12. 22%
19%
18%
7%
7%
6%
5%
4%
4%
8%
Adenovirus
Retrovirus
Naked/Plasmid DNA
Adeno-associated Virus
Vaccinia Virus
Lentivirus
Lipofection
Poxvirus
Herpes Simplex Virus
Other Vectors
Unknown
Figure: Vectors used in gene therapy.

13. 0
2
4
6
8
10
12
14
16
18
20
Figure : Gene type transferred in gene therapy .

14. 0 20 40 60 80 100 120 140 160 180
2016'
2014'
2012'
2010'
2008'
2006'
2004'
2002'
2000'
1998'
1996'
1994'
1992'
1990'
Unknown
Figure: Approved worldwide data of gene therapy.

15. CONCLUSION
 The necessity of gene therapy is inevitable
in treating genetic disorders for which
there is no permanent cure.
 Scientists are more focused on treating
cancer by means of gene therapy although
other disorders demand attention
 USA is now the leader in researches
related to gene therapy

16. CONCLUSION
 There is no unique vector of choice. This
choice is dependent on many different
factors.
 Inflammatory gene insertion is becoming
increasingly popular
 Despite the tedious efforts of over 50 years
still most gene therapy trials didn’t
produce successful fruit.
 No. of clinical trials associated with gene
therapy is rising up.

17. CONCLUSION
 No papers have been published in
Bangladesh regarding gene therapy.
 Bangladesh has the potential to ride on
the vehicle of gene therapy research.

18. REFERENCES
1. a D. Miller, “Human gene therapy comes of age.,” Nature, vol. 357, no.
6378, pp. 455–460, 1992.
2. T. Friedman, “A brief history of gene therapy,” Nature Genetics, vol. 2, no.
2. pp. 93–98, 1992.
3. W. S. M. Wold and K. Toth, “Adenovirus vectors for gene therapy,
vaccination and cancer gene therapy.,” Curr. Gene Ther., vol. 13, no. 6, pp.
421–33, 2013.
4. Gene Therapy Clinical Trials Worldwide Provided by the journal of
Gene Medicine. http://www.abedia.com/wiley/index.html.
5. D. R. Huddart, “Gene therapy hope for allergies and asthma,”
BioNews, 2017. [Online]. Available :
http://www.bionews.org.uk/page_849095.asp.

19. THANK
YOU