1. Dr. APJ ABDUL KALAM TECHNICAL UNIVERSITY
(Formerly Uttar Pradesh Technical University) LUCKNOW
Noida Institute of Engineering & Technology
(Pharmacy Institute)
Project Title : Gene Therapy
Submitted To:
Dr. Saumya Das
Associate Professor
Noida Institute of Engineering &
Technology(Pharmacy Institute)
Greater Noida
Submitted By:
Shilajit Das
M. Pharm(Pharmacology)
Noida Institute of Engineering &Technology
(Pharmacy Institute) Greater Noida
2. Gene Therapy - Introduction
▪ Gene therapy is the introduction of genes into existing cells to
prevent or cure a wide range of diseases.
▪ It is a technique for correcting defective genes responsible for
disease development.
▪ The first approved gene therapy experiment occurred on September
14, 1990 in US, when Ashanti DeSilva was treated for ADA-SCID.
3. IN VIVO GENE THERAPY
▪ Direct delivery of therapeutic gene into
target cell into patients body.
▪ Carried out by viral or non viral vector
systems.
▪ It can be the only possible option in
patients where individual cells cannot
be cultured in vitro in sufficient
numbers (e.g. brain cells).
▪ In vivo gene transfer is necessary when
cultured cells cannot be re-implanted in
patients effectively. Ref : shorturl.at/tzUXY
4. EX VIVO GENE THERAPY :-
Transplant the modified cells to the patient.
Select genetically corrected cells and grow.
Introduce the therapeutic genes.
Grow the cells in culture
Isolate cells with genetic defect from a patient
5. Other types of gene therapy
GENE AUGMENTATION THERAPY
▪ Most common form of gene therapy.
▪ Foreign gene replaces missing or defective gene.
▪ Eg. Replacement of defective p53 gene by a normal one in liver cancer.
GENE INHIBITION THERAPY
▪ Done to block the overproduction of some proteins.-
2 types - Antigene and antisense therapy.
- Antigene-blocks transcription using antigene oligonucleotide.
- Antisense-blocks transalation using antisense oligonucleotide.
6. VECTORS IN GENE THERAPY
▪ To transfer the desired gene into
a target cell, a carrier is
required. Such vehicles of gene
delivery are known as vectors.
2 main classes
- Viral vectors.
- Non viral vectors
Ref : shorturl.at/fvSZ0
7. VIRAL VECTORS IN GENE THERAPY
▪ ADENO VIRUS VECTOR SYSTEM
▪ Adeno virus with at DNA
genome - good vectors.
▪ Target- non dividing human cell.
▪ Eg. Common cold adenovirus.
8. NON VIRAL VECTOR SYSTEM
PURE DNA CONSTRUCT
▪ Direct introduction of pure DNA construct into target tissue.
▪ Efficiency of DNA uptake by cells and expression rather low.
▪ Consequently, large quantities of DNA have to be injected periodically.
LIPOPLEXES
▪ Lipid DNA complexes: DNA construct surrounded by artificial lipid layer.
▪ Most of it gets degraded by lysosomes..
9. Factors to be considered in Gene therapy
▪ How to deliver genes to specific cells, tissue and whole animals?
(methods of delivery)
▪ How much and how long the introduced gene will be expressed?
▪ The site and dose of gene delivery
▪ Is there any adverse immunological consequence of both delivery
vehicle (Virus) and the gene in animals?
▪ Is there any toxic effects?
10. Uses of gene therapy
▪ Clinical gene transfer applications
▪ Vaccine development
▪ Production of transgenic animals
▪ Treatment of cancer and AIDS
▪ Gene discovery
▪ Gene therapy
▪ Enhancing the resistance of plant
▪ Genetically modified organism
11. Application of gene therapy
▪ Genetic disorder (deficiency): OTC
▪ Cancer
- Genetic predisposition
- Mutation in oncogene or tumor suppressor gene
▪ Autoimmunity diseases: rheumatoid arthritis
- Delivery of counteracting gene
▪ Diseases involve several genes and the environmental interact:
diabetes
12. CONCLUSION
▪ Theoretically, gene therapy is the permanent solution for genetic
diseases.
▪ But it has several complexities. At its current stage, it is not
accessible to most people due to its huge cost.
▪ A breakthrough may come anytime and a day may come when
almost every disease will have a gene therapy.
▪ Gene therapy have the potential to revolutionize the practice of
medicine.
13. REFERENCES
▪ Dubey R.C, A textbook of biotechnology, 1st edition (2004), S Chand
and company, New Delhi
▪ Gupta P.K, Elements of Biotechnology, 1stedition (2001), Rastogi
Publications, Meerut.
▪ Satyanarayana U, Biotechnology, 1st edition, Book andallied (P) Ltd,
Kolkata
▪ http://www.medindia.net/articles/genetherapy_treat ment.htm
▪ http://en.wikipedia.org/wiki/Gene_therapy
▪ shorturl.at/tzUXY
▪ shorturl.at/fvSZ0
