Gene therapy drug delivery system (1).pdf

Gene therapy drug delivery system
Gene therapy-Gene therapy is a technique which is replacing a functional gene with non functional gene.
Gene therapy involves one or more foreign gene into an organism to treat hereditary or acquired genetics defects
Gene therapy utilize the transfer of genetic information for modification of a phenotype for therapeutic response.
Classical gene therapy:-It involve therapeutic gene delivery and optimum expression inside the target cells.
The foreign bodies gene carry out following function.
Produce a product(protein)that the patient lacks.
Produces toxin so that diseases cell is killed.
Activated cell of the immune system so as to help in killing of diseases cell.

1. Biotechnology
By Manish Singh
Assistant Professor
Sai college of pharmacy

2. Gene therapy drug delivery system
Gene therapy-Gene therapy is a technique which is
replacing a functional gene with non functional gene.
Gene therapy involves one or more foreign gene into an
organism to treat hereditary or acquired genetics
defects
Gene therapy utilize the transfer of genetic information
for modification of a phenotype for therapeutic
response.

4. Types of gene therapy
Somatic cell
gene therapy-
Therapeutic gene
transferred into somatic
cells
Eg:-Introduction of gene
into bone marrow cells,
blood cells, skin cells
At present all researchers
directed to correct
genetic defect in
somatic cells.
Germ line gene
therapy-
Therapeutic gene transferred into
the germ cells
Eg:-Gene introduced into eggs
and sperm cells
It is inheritable and passed on to
later generation
For safety ,ethical and technical
reason, it is not being attempted
at present.

6. Gene therapy approaches
Classical gene therapy:-It involve therapeutic
gene delivery and optimum expression inside the target cells.
The foreign bodies gene carry out following function.
Produce a product(protein)that the patient lacks.
Produces toxin so that diseases cell is killed.
Activated cell of the immune system so as to help in killing of diseases cell.

7. Non-classicalgene therapy
It involve the inhibition of gene expression
associated with the pathogenesis or correct a
genetic defect and restore the normal gene
expression.

9. Method of Gene therapy
There are mainly two approachesfor the transfer
of gene in gene therapy
Ex-vivo gene therapy:-Transfer of gene
into patient cells outside the body.
In vivo gene therapy:-Transfer of gene
directly into cells inside the body.

11. How to work gene therapy?

12. Vector for gene therapy
There are two types of vector of gene therapy.
Viral vectors for gene therapy:-It is recognized as powerful strategy for
gene therapy.
Some of the important viral includes-Retro virus, Adeno virus, Adeno
associated virus.
Non-viral vectors-It uses a naked DNA or DNA complexes.
Eg:-Oligonucleotides, Naked DNA , Liposomes, Micro injection, Gene gun
delivery , Hybrid method, Chemical method etc

14. Advantage of gene therapy
It can be used to treat genetic disorder like colour blindness.
It can be used to treat sickle cell diseases.
It can be used to treat haemophilia.
It can be used to treat dwarf which are causes by deficiency of growth
hormone.
It can be used to treat diabetes disorder which are due to low secretion of
insulin in the body.
It can be used to treat cancer disorder like brain cancer, lung cancer ,
breast cancer and blood cancer.

15. Disadvantage of Gene therapy
Unwanted immune system reaction.
Targeting the wrong cell.
Infection cause by virus.
Possibility of causing a tumour.

16. Application of Gene therapy
Vaccine development
Clinical gene transfer application.
Production of transgenic animals
Treatment of cancer and AIDS.
Gene discovery.
Enhancement of resistance of plant.
Genetically modified organism.

17. Reference
www.fda.gov.
Slide share of strategies of gene therapy.
www.nhlbi.nih.gov.
Gene therapy-Stellar iasacademy.
Embryo editing gene.
frontiersin.org
researchgate.net
www.mayoclinic.org