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Aditi Birole 1
Gene Therapy:-
A topic to be Known…
20/03/2010
Aditi Birole 220/03/2010
Contents:-1. Introduction2. Overview3. Basic Functions4. Characteristics5. Functioning6. Gene Therapy7. Disadvantages of Gene therapy8. Conclusion9. References
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Introduction:-20/03/2010
Gene Therapy :-The introduction of nucleic acids into cells for the purpose
of altering the course of medical condition or disease.The basic unit of genetic information
They determine the nature and the function of the cell.The human genes (about 140,000) are referred to as the human genome.A genome is the full set of genes in each cell of an organism.
E.g.:- Administration • ex vivo- cells removed, genetically modified, transplanted back into a patient
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Some Facts About Genes:-
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Genes Plantation:-20/03/2010
• High concentration of virus allowing many cells to be infected or transduced
• Convenience and reproducibility of production
• Ability to transduce dividing and non-dividing cells
• Ability to integrate into a site-specific location in the host
chromosome, or to be successfully maintained as stable episome
• A transcriptional unit that can respond to manipulation of its
regulatory elements
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References:-Books:-
1. Genes Therapy
Websites:-1. www.google.co.in2. www.wikipedia.com3. www.genetics.com
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Cystic Fibrosis
Blood Disorders
Muscular Dystrophy
Diabetes
Genetic Diseases:-
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Gene formation
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The DNA:-
20/03/2010
The substance that genes are composed of that determines the nature of each cell and how it will function.
The arrangement of these pairs along the DNA molecule determines which kind of proteins that will be formed in the cell.
10
What Do You Think?
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METHODS MAJOR LIMITATIONS
1. Calcium Phosphate
DEAE Dextran Low Efficiency
Cationic Lipids, Liposomes
2. Direct DNA Injections Low Efficiency
3. Electroporation Transient expression
Research Says:-
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liver muscle
CMV promoter Rhodopsin promoter
retina brain
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Conclusion:- With the above study I found that the genetic
therapy is hot topic to research and promising effort have been brought to yield awesome results.
Genetic therapy has some drawback which can removes on further experiments.
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Thanking You…….
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Any Questions:-