GENE THERAPY

Shaping the Future.

Meshal Cheema(10I-1977)
Hajira Uzair(19I-0737)
CONTENTS

 What is gene therapy?

 Target
 Strategy
 Clinical trials
 Hurdles
 Some facts
 Conclusion
What is gene therapy?
 conceptualized in 1972.
 Human gene transfer.
 The Therapeutic delivery of nucleic acid as a drug.
 Somatic cell gene therapy.
 Germ line gene therapy.
 First attempt (1980 by Martin Cline)
 First successful nuclear gene transfer (1989)
 First successful therapeutic gene transfer (14 september
1990)
 Ashi Desilva treated for ADA-SCID
TARGET
 Killing of specific cells, cancer etc

 Production of healthy gene

 Eliminating inherited diseases

 Curing the defective gene

 People can easily live a long life

STRATEGY
 Creating DNA in laboratory
 Inserting DNA into gene via vectors.
 Viral vectors (recombinant viruses).
 Non viral vectors (naked DNA)
 Inserted in nucleus
 Becomes a part of cell
 Starts to produce new protein
CLINICAL TRIALS

 Over 2930 trials completed

 31 countries
 America (63.5%)
 Europe (28.3%)
 Asia (3.4%)
 HURDLES
 Short lived nature
 Immune response
 Viral vectors
 Insertional mutagenesis
 Cost
CONCLUSION
 Potential to cure numerous diseases.

 Minimal risk to patient

 Efficiency is increasing

 Will open a new door for genetic enigineering

 Designer babies