The document discusses nucleic acid-based therapeutic delivery systems, focusing on gene therapy as a method to treat genetic and acquired disorders by inserting new genetic material into cells. It outlines the types of gene therapy, including germline and somatic cell therapy, as well as the processes and methods of delivery using viral and non-viral vectors. The conclusion emphasizes the potential of this technique to treat various diseases, prevent genetic disorders in future generations, and improve the overall understanding of gene functions.

1. NUCLEIC ACID BASED THERAPEUTIC
DELIVERY SYSTEM
By-
Parthajeet Kalita* &
Pervej Alom Barbhuiya
M.Pharm 2nd sem
Faculty of Pharmaceutical Science
Assam Down Town University
Panikhaiti, Ghy-26, Assam

2. CONTENTS
• Introduction
• Gene therapy
• Types of gene therapy
• Germline gene therapy
• Somatic cell gene therapy
• Basic process of gene therapy
• Potential target diseases for gene
therapy
• Conclusion

3. INTRODUCTION
• The delivery of nucleic acid molecule into cell to
alter physiological functions at the genetic level is a
powerful approach to treat a wide range of inherited
and acquired disorders.
• This technique has been a common research tool
in the laboratory for decades to study gene
functions.
• The therapeutic potential of this approach was not
fully realized due to lack of reliable and practical
methods to transfer and express recombinant DNA in
mammalian cells
• By the 1980s a series of clinical trials
demonstrated therapeutic efficacy from the
transplantation of virally transduced cell.

4. GENE THERAPY
• Insertion of new genetic material into the cells of
an individual with the intention of producing a
therapeutic benefit for the patient is human gene
therapy.
• Gene therapy quickly became an intensely
investigated field with the promising potential to
devise treatment not only for genetic diseases but
also for a wide range of disorders including
metabolic disorders, infections disease, chronic
illness and cancer.
• The power of gene therapy is derived from the
ability to manipulate cell physiology at genetic
and epigenetic levels.

5. TYPES OF GENE THERAPY
Gene
Therapy
Germ Line
Gene
Therapy
Somatic
Gene
Therapy

6. GERM LINE GENE THERAPY
• In germ line gene therapy germ cells
are modified by introduction of
therapeutic genes.
• Modifying a germ cell causes all the
organism cells to contain the modified
gene.
• The change is therefore heritable and
passed on to later generations.
• For safety ethical and technical reasons,
it is not being attempted at present.

7. SOMATIC CELL GENE THERAPY
• In somatic cell gene therapy, the
therapeutic genes are transferred into any
cell other than a germ cell.
• Introduction of genes into bone marrow
cells, blood cells, skin cells.
• It will not be inherited to later generations.
• Most focus on severe genetic disorders,
including immuno deficiencies, hemophilia,
thalassaemia, and cystic fibrosis. Such
single gene disorders are good candidates
for somatic cell therapy.

8. TYPES OF SOMATIC CELL GENE
THERAPY
Ex Vivo
Can be applied
to selected
tissues
The direct delivery
of the therapeutic
gene into the target
cells
Cells are
modified
outside the body
and
transplanted
back
Cells are modified
inside the body
and transplanted
back
In Vivo

9. EX VIVO GENE THERAPY
Isolate cells with genetic defect from a patient
Grow the cells in culture
Introduce the therapeutic genes
Select genetically corrected cells and grow
Transplant the modified cells to the patient

10. 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.
 In vivo gene transfer is necessary when cultured
cells cannot be re implanted in patients effectively.

11. BASIC PROCESS OF GENE THERAPY
Viral vector
 Viruses have evolved a way of encapsulating and
delivering their genes to human cells in a pathogenic way
by manipulating the viral genome to remove disease
causing gene and inserting therapeutic one.
 Viruses bind to their hosts and introduce their genetic
material into the host cell.
 Viruses used are altered to make them safe,
although some risks still exist with this type of gene
therapy.

12. TYPES OF VIRAL VECTORS
 Retrovirus vector system
 Adeno virus vector system
 Adeno associated virus vector
 Herpes simplex virus vector

13. NON VIRAL VECTOR
 The non viral vectors are naked DNA, particle
based and chemical based.
 They are administered by direct administration.
 Most of cardiovascular clinical trials use non viral
vectors as a mode of gene transfer.
 Non viral vectors are generally used to transfer
following types of nucleic acids
1. Small DNA
2. Large DNA
3. RNA

14. METHODS OF NON VIRAL VECTOR GENE
DELIVERY
Needle Injection Physical Methods-
 Electroporation
 Gene Gun
 Hydroporotion
Chemical methods-
Inorganic particles
Synthetic/Natural biodegradable
polymers
 Chitosan

15. POTENTIAL TARGET DISEASES FOR GENE
THERAPY
GENETHERAPY FORTREATMENT OF GENETIC
DISORDERS:-
 Genetic disorders are illness stemming from
errors in a person’s genes
 Any mistake in a gene can alter how a specific
protein is produced.
 Without proper proteins, the body will not function
properly and will take on a chronic and possibly life
threatening condition.
 Genetic disorders can be congenital meaning they
occur from birth or they may develop over time.

16. GENE THERAPY CURES BLINDNESS
Cures blindness of inherited condition
Inherited blindness caused by an abnormally in a gene
called RPE65.
The condition appears at birth or in the first few
months of life and causes progressive worse and loss
of vision.
It can be treated by enabling access to cells beneath
the retina of patients.

17. GENE THERAPY FOR CANCER TREATMENT
 ONCOGENE ACTIVATION
Done by using antisense therapy
 Reduces the expression of
antigenic proteins responsible for
malignancies
 VIRUS MEDIATED ONCOLYSIS
 Adeno virus and herpes virus can
infect and cause lysis of cancer
cells.
 AUGMENTATION OF TUMOR SUPRESSOR
GENE
 It is done by repair of tumor suppressor
gene in malignant cells.

18. GENE THERAPY IN SOME OTHER DISEASES
 Gene therapy can also used in other diseases like-
Diabetes
Cystic fibrosis
Parkinson’s disease
X-SCID
Adenosine deaminase deficiency
Muscular dystrophy

19. CONCLUSION
Nucleic acid based therapeutic delivery is a
technique that will be very useful for the treatment
of many diseases.
Gene therapy can be helpful in many genetic
diseases which can prevent those diseases for future
generations.

20. THANK YOU