TREATMENT OF DM BY USING GENE THERAPY

1. HUMAN GENE THERAPY
PRESENTED BY
MR. VILAS KAMBLE
GUIDED BYGUIDED BY
DR. (MRS.) USHA SHINDEDR. (MRS.) USHA SHINDE
PADMASHREE DR D.Y. PATIL COLLEGE OFPADMASHREE DR D.Y. PATIL COLLEGE OF
PHARMACY AKURDI PUNE-411033PHARMACY AKURDI PUNE-411033
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2. CONTENT
 INTRODUCTION
 BRIF HISTORY OF GENE THERAPY
 APPROCHES FOR GENE THERAPY
 STAPES INVOVED IN GENE THERAPY
 TYPES OF GENE THERAPY
 VECTOR USED IN GENE THERAPY
 EXAMPLES OF DISEASE
 BRIF ABOUT SCID
 CURRENT SINARIO OF GENE THERAPHY FOR
DIABETES MELLITUS
 TREATMENT OF DM BY USING GENE THERAPHY
 USES OF GENE THERAPHY
 REFERENCES
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3. INTRODUCTION1
Advance in biochemistry and molecular
biology have helped to uderstand the genetic
basis of disease.It was dream of the researchers
to replace the defective genes with good once,and
cure the genetic disorder.1
Defination:-
Gene therapy can be broadly defined as the
transfer of genetic material to cure a disease or
at least to improve the clinical status of a
patient.2
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4. BRIF HISTORY OF GENE
THERAPY3
Sr
no.
Year Scintist name Work done
1 1972 Fridman and
Robin
Peblished a paper in Science
titled Gene therapy for human
genetic disease.
2 1972 Rogers was cited for proposing that
exogenous good DNA be used to
replace the defective DNA in
those who suffer from genetic
defects
3 1990 William French
Anderson
31 Dec-1936
Pionear of gene theraphy.
He success in gene therapy of
Four-year-old Ashanti DeSilva
received treatment for a genetic
defect that left her with SCID.
(14 Sep 1990)
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5. APPROCHES FOR GENE
THERAPY
 Insertion of normal gene inplace nonfunctional gene.2
 Repairing of abnormal genes.2
 Somatic cell gene theraphy.1
 Germ cell gene theraphy.1
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6. STAPES INVOVED IN GENE
THERAPY1
Invitro experiment and research on lab animal
(pricliical trial)
Phase-I Trial with a small no of human subject.
Phase-II Trial with more human subject to assess
whether the produt is helpful or not.
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7. STAPES INVOVED IN GENE
THERAPY1
Phase-III trial in larg human sample for final and
compreesive analysis of the safety and efficacy of
the product
Final therapy or product launch in the market
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8. TYPES OF GENE THERAPY1
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9. TYPES OF GENE THERAPY1
EX-VIVO GENE
THERAPHY
IN-VIVO GENE
THERAPHY
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10. VECTOR USED IN GENE THERAPY
 Vector:-
The carrier partical or molecules used to
deliver gene to somatic cells are called vector.1
 Types of vector:-
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11. VECTOR USED IN GENE THERAPY
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12. EXAMPLES OF DISEASE1
 Selected list of genetic disease that are likely to
be cure by using bone marrow cells are-
 SCID
 Sickle cell anemia
 Thalassemia
 Hunter disease
 Osteoporesis
 Diabetes
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13. BRIF ABOUT SCID
 It also know as alymphocytosis or Glanzmann–Riniker
syndrome.4
 The most commonly quoted figure for the prevalence of
SCID is around 1 in 100,000 births.4
 Human SCID is a prenatal disorder of T lymphocyte
development, already present at birth even if clinically
silent in most affected newborns.5
 This is rare inherited disorder associated with T-
lymphocytes disfunction.5
 About 50% of SCID patient have defect in the gene located
on chromosomes 20,that encode for adenosine diaminase.5
 No production of adenosine diaminase5
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14. BRIF ABOUT SCID1
No production of adenosine diaminase.
Accumulation of deoxyadenosine and it’s
metabolites deoxyadenosine-5-phosphate
Accumulated metabolite destroyed T-lymphocytes
present in the body
Collaped the immunity system of the body
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15. BRIF ABOUT SCID
Persone suffer for various infective diseaseses
Die at the young age
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16. TREATMENT OF SCID BY GENE
THERAPY
Universal blue circleUniversal blue circle
symbol for diabetessymbol for diabetes..
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17. CURRENT SINARIO OF GENE
THERAPHY FOR DIABETES MELLITUS
 Diabetes mellitus (DM), commonly referred to
as diabetes, is a group of metabolic diseases in
which there are high blood sugar levels over a
prolonged period6
 Epidemiology:-
Acco. International Diabetis Fedration,
1.One peaple of DM per two normal
peaple.
2.4.9 million people death because of DM
untill 2014.
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18. BRIF HISTORY OF DM
 First diseases described, with an Egyptian
manuscript from at 1500 BC
 Indian physicians around the same time
identified the disease and classified it as
madhumeha.(honey urine).
 Diabetes=to pass through
 Hippocrates said that, “DM is incurable”
 Galen named the disease “diarrhea of the urine”
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19. WHAT IS DM8
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20. TREATMENT OF DM BY USING GENE
THERAPHY 10
 Treatment of type-I DM
Type-I DM caused by body own immune cell
damage of b-cell
Uses chord blood to reeducate DM Own cell
regulate pancreatic function
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21. USES OF GENE THERAPHY
 Disease name
 SCID
 Sickle cell anemia
 Thalassemia
 Hunter disease
 Osteoporesis
 Diabetes
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22. PROBLEMS IN GENE
THERAPY1,9,10
 Short-lived nature of gene therapy
 Immune response and toxicity
 Problems with viral vectors
 Restricted targeting of specific cell types
 Multigene disorders
 Insertional mutagenesis
 Religious concern
 Deaths may occured
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23. REFERENCES
1.Satyanarayana U., “Biotechnology”,published by
books and allied Pvt.Ltd,eight edition 2013,page no-
157,
2.Patil P.M, et al,Review Article On Gene
Therapy,International Journal of Genetics ISSN: 0975-
2862 & E-ISSN: 0975-9158, Volume 4, Issue 1,
2012,:74-79.
3.https://en.wikipedia.org/wiki/Gene_therapy#cite_note-
55
4.https://en.wikipedia.org/wiki/Severe_combined_immu
nodeficiency
5. Cossu F. Italian Journal of Pediatrics 2010, 36:76.
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24. REFERENCES
6. https://en.wikipedia.org/wiki/Diabetes_mellitus.
7. Ozougwu, J. C. , Obimba, K. C. , Belonwu, C.
D.3 , and Unakalamba, C. B, Review The
pathogenesis and pathophysiology of type 1 and
type 2 diabetes mellitus , Journal of Physiology and
Pathophysiology, Vol. 4(4), pp. 46-57, September,
2013 DOI 10.5897/JPAP2013.0001 ISSN 2I41-
260X © 2013 Academic Journals
8. http://www.idf.org.
9. https://en.wikipedia.org/wiki/History_of_diabetes.
10.Patel K.P,Joshi H.M,Majumdar E.P,Patel
V.J,Newer approches in tretment of DM,NHL journal
of medical science,Jan-2013,vol-2,issue-I.
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