important ppt for m pharma pharmacology student

1. ADVANCE COLLEGE OF PHARMACY
Kanpur
DEPARTMENT OF PHARMACOLOGY
GENE THERAPY
ANUBHAV DUBEY
M.Pharma
pharmacology

2. CONTENTS
 INTRODUCTION
 TYPES OF GENE TRANSFER TECHNIQUE
 Method of gene delivery
 ADVANTAGE
 DISADVANTAGE
 APPLICATION
 CUNCLUSION

3. •It is a technique for correctiing defective gene
responsible for disease development
•Gene therapy is the introduction of gene into existing
cell to provide or cure wide range of disease
•Researcher may used one of the several approaches
for correcting faulty gene …..
Flow chart of gene therapy
Introduction

4. • A normal gene may be inserted into non specific
location with the genome to replace nonfunction
gene
• An abnormal gene could be swapped for normal
gene recombination .
• The abnormal gene could be replace through
selective reverse mutation which return the
gene to normal function

5. Types of gene transfer technique
•To transfer the desired gene into a target cell a
carrier is required such vehicles of gene delivery
are known vector.
Two main classes vector
Viral vectors Non viral vectors
Ritro virus vector system
Adenovirus vector system
Adeno associated virus
vector
Herpex simplex virus
vector
Pure DNA construct
Lipolex
DNA molecule conjugation
Human artificial
chromosome

6. Viral method of gene therapy

7. Ritro virus vector system
retrovirus have the ability to integrate into host genome in a
stable fusion …
.1can carry DNA of size less than 3.4kb.
.2 replication defective virus particle .
.3 target cell dividing.
Adenovirus vector system
Adenovirus with DNA genome good vector
target –Non dividing human cell .
Eg. common cold virus
Adeno associated virus vector
.1It is a human virus that can integrate into chromosome 19.
.2 it is a single standard non pathogenic small DNA virus.
.3Adeno associated virus vector enter host cell become
double

8. Herpex simplex virus vector
. 1 they infect particular type of cell.
.2 they infect and persist in nervous cell
Non viral vectors
Pure DNA construct
in this method direct introduction of pure DNA construct into
target tissue consequently large quantities of DNA have to be
inject periodically
lipolex
lipid DNA complex DNA construct surround by artificial
lipid layer most of its degrade by liposome .
DNA molecule conjugation
commonly used synthetic conjugated is poly L lysine bound
to specific target cell receptor .
it avoids lissome breakdown of DNA.

9. Human artificial chromosome
. can be carry large DNA that is with one or more
therapeutic with regulatory elements
Liposome for drug delivery

10. Method of gene delivery
1.chemical method
.using detergent mixture
.Lipo protein .
2.phyical method
.gene gun
.microinjection
using detergent mixture
chemical compound such as calcium phosphate are mixed
with functional CDNA of desired function
lipoprotein
Liposome are artificial phospholipids vehicle used to
determine variety of molecule including DNA Into the cell

11. Delivery of gene by direct and cell based

12. Advantage
.Gene therapy has the potential to eliminate and prevent
heredity disease such as cystic fibrosis ADA –SCID etc.
. It is possible cure heart disease ,AIDS, and cancer .
.TO treat genetic disease.
.It is helpful to identification cardiac disease ,from the
future generation .
Disadvantage
.short lived nature of gene therapy .
.immune response –genes injected with virus may
trigger on immune response against the virus .
. Mutagenic disorder

13. Application
• In case of Parkinson's diseases
•In case of Alzheimer's Diseases
•In case of diabetics Neuropathy
•In case of monastic melanoma

14. Conclusion
According to gene therapy different types of genetic
disorder are cured. In case of cystic fibrosis, Diabetes,
AIDS, Hepatitis melanoma, Alizhmer, Parkinson’s diseses
etc.
In case of Parkinson’s disease one trial is done
Neurologix a biotech company announced that they have
successfully completed its landmark Phase I trial of gene
therapy[] for Parkinson's Disease
12 patient study with four patients in each of three dose
esca-lating cohorts. All procedures were performed under
local anesthesia and all 12 patients were discharged from
the hospital within 48 hours of the procedure, and followed
for 12 months. Primary outcomes of the study design,
safety and tolerability, were successfully met. There were

15. Steps involved in treatment of Parkinson’s disease ..
In case of Diabetes gene therapy also play a good role.
Re-placing a mutated gene that causes disease with a
healthy
copy of the gene.
Inactivating, or “knocking out,” a mutated gene that is
func-tioning improperly.
Introducing a new gene into the body to help fight a
disease.