nucleic acid and gene therapy

1. Pharmaceutical role of nucleic acid

2. LaraibTariq
PharmD
1st semester

3. Pharmaceutical
role of nucleic acid

4. Pharmaceutical
Importance Of
Nucleic Acid
૱ Used in cancer, cardiovascular and autoimmune disease.
૱ The pharmaceutical perspectives of Nucleic acid based
therapy presents a comprehensive account of gene
therapy.
૱ Control gene regulation, transcription, translation and
replication.

5. It is a technique for correction of genetic deficiency in a cell
by the addition of new DNA to the cell. This definition has
been expanded to include treatment of acquired disease by
addition of new DNA.
There are four approaches:
₠ A normal gene inserted to compensate for nonfunctional
gene.
₠ An abnormal gene inserted for normal function.
₠ Change the regulation of gene pairs.
Gene
Therapy

6. Gene
Therapy
Strategies
£ Gene Augmentation Therapy(GAT)
£ Gene inhibition Therapy
£ Gene replacement Therapy
£ Gene correction
£ Targeted killing of specific cells
£ Prodrug Therapy

7. Types Of
Gene
Therapy
Two types of gene therapy
¤ Somatic Gene Therapy: involves introducing a “good”
gene into targeted cells with the end result f treating the
patient not the offspring.
¤ Germline Gene Therapy: involves modifying the genes in
egg or sperm cells ,which will then pass any genetic
changes to next generation.

8. Somatic
Gene
Therapy
Three categories of somatic cell gene therapy :
1. Ex-vivo : cells removed from body ,incubated with
vector and gene engineered cells returned to body.
2. In-situ : vector is placed directly into affected tissue.
3. In-vivo : vector is injected directly into blood stream.

9. Application
of Gene
Therapy
◊ Cancers
◊ Inherited disorders
◊ Infectious diseases ( viral or bacterial)
◊ Immune system disorders
◊ Vaccination
cancer
Diabetes
Parkinson

10. CancerApproaches
∂ Delivery of genes encoding toxic molecules to cancers
cells to kill them.
∂ Delivery of genes encoding chemokines to cancer cells to
activate the immune response to kill them.
∂ Antibody therapies: DNA vaccines with genes that encode
antibodies to cancer specific proteins in tumor cells
∂ Insertion of normal tumor suppressor genes into cells.
∂ Antisense therapy: DNA that blocks the synthesis of
proteins encoded by deleterious genes.

11. Problems
with Gene
Therapy
€ Short lived.
€ New things introduced leads to immune response.
€ Patient could have toxic, immune, inflammatory response.
€ May cause disease once inside.
€ Multi gene disorders.
€ Heart disease, high blood pressure, Alzheimer,s, arthritis
and diabetes are hard to treat because you need to
introduce more than one gene.

12. Requirement
of Gene
Therapy
π Understanding of disease process.
π Structure/function of gene to be introduced.
π Efficient delivery of gene.
π Control of gene expression.
π Prevention/control of immune response.
π Animal model and assessment of function.
π Clinical trial.