Gene Therapy 2) What is Gene Therapy An approach of treating diseases by either modifying the expressions of an individual’s genes or correction of abnormal genes. 3) This can be accomplished by: Replacing a mutated gene that causes disease with a healthy copy of the gene. Inactivating, or “knocking out,” a mutated gene that is functioning improperly. Introducing a new gene into the body to help fight a disease 4) NON VIRAL VECTOR SYSTEM 5) 1. 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. 6) 2. DNA MOLECULAR CONJUGATES Commonly used synthetic conjugate is poly- L- lysine bound to specific target cell receptor. Therapeutic DNA is then made to combine with the conjugate to form a complex. It avoids lysosomal breakdown of DNA 7) 3. LIPOPLEXES Lipid DNA complexes DNA construct surrounded by artificial lipid layer. Most of it gets degraded by lysosomes 8) 4. HUMAN ARTIFICIAL CHROMOSOME Can carry a large DNA i.e., with one or more therapeutic genes 9) References :- Biotechnology by U. Satyanarayana and U. Chakrapani, First edition, books and allied (p) Ltd,2005. Page no.213-226. NPTEL-Bio Technology – Genetic Engineering and applications “MODULE 5-LECTURE 1 GENE TRANSFER TECHNIQUES : BIOLOGICAL METHODS .” Page no : 3-22.

1. Gene Therapy
(Non Viral Gene Transfer )
 PRESENTED BY 
MR.Shrikant R. Divekar
(M.PHARM FIRST YEAR)
(503)
P H A R M A C E U T I C S
Guided By
Dr.(Mrs.) S. Chaudhari 1

2. An approach of treating diseases by either
modifying the expressions of an individual’s
genes or correction of abnormal genes.
2

3. This can be accomplished by:
*Replacing a mutated gene that causes disease with a
healthy copy of the gene.
*Inactivating, or “knocking out,” a mutated gene that is
functioning improperly.
*Introducing a new gene into the body to help fight a
disease
3

4. NON VIRAL VECTOR SYSTEM
4

5. 1. 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.
5

6. 2. DNA MOLECULAR CONJUGATES
*Commonly used synthetic conjugate is poly- L- lysine bound
to specific target cell receptor.
*Therapeutic DNA is then made to combine with the conjugate
to form a complex.
*It avoids lysosomal breakdown of DNA
6

7. 3. LIPOPLEXES
*Lipid DNA complexes
*DNA construct surrounded by artificial lipid layer.
*Most of it gets degraded by lysosomes
7

8. 4. HUMAN ARTIFICIAL CHROMOSOME
*Can carry a large DNA i.e., with one or more therapeutic
genes
8

9. References :-
Biotechnology by U. Satyanarayana and U.
Chakrapani, First edition, books and allied (p)
Ltd,2005. Page no.213-226.
NPTEL-Bio Technology – Genetic Engineering and
applications “MODULE 5-LECTURE 1 GENE TRANSFER
TECHNIQUES : BIOLOGICAL METHODS .” Page no : 3-
22.
9