Name of the title: Nucleic Acid-Based Therapeutic Delivery System. It includes information about nucleic acid, gene therapy, and its type, a method to deliver the desired DNA, i.e., vectors and their types, with proper examples and diagrams, and how these things help in delivering a nucleic acid-based therapeutic drug delivery system.

1. Made By :- Pramesh Panwar

2. Nucleic acids are large biomolecules that are crucial in all cells and viruses. A major
function of nucleic acids involves the storage and expression of genomic
information. They are composed of nucleotides, which are the monomer components: a
5-carbon sugar, a phosphate group and a nitrogenous base. The two main classes of
nucleic acids are deoxyribonucleic acid and ribonucleic acid.
WHAT IS NUCLEIC ACID?

3. NUCLEIC ACID BASED THERAPEUTIC
DELIVERY SYSTEM ?
The delivery of nucleic acid molecule into cells to alter physiological functions at the the
genetic level is a powerful approach to treat a wide range of inherited and acquired
disorders.
This technique Study gene function
Help in
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.

4. GENE THERAPY
•Insertion of new genetic material into the cells of an individual with the intention of
producing a therapeutic benefits for the patient is human gene therapy.
•The power of gene therapy is derived from the ability to manipulate cell physiology
at genetic and epigenetic levels.
•There are more than 3000 genetic disease in humans. Only few of them are treatable.
Ex:- phenylketonuria, hemophilia-A, cystis fibrosis, severe combined
immunodeficiency (SCID), emphysema, hyper-ammonia, sickle celll anaemia etc
•On Sep 14, 1990 researchers at the U.S. National institute of health performed the
first (approved) gene therapy procedure on 4y old Ashanti DeSilva. She had a rare
genetic diseased called severe combined immune deficiency (SCID) because of a
defective gene called adenosine deaminase (ADA), Therefore, she lacked a healthy
immune system and was vulnerable to every germs.

5. In Ashanti’s gene therapy procedure, doctors remove white blood cells from the
child’s body, grew the cells in the laboratory, inserted the missing gene into the
cells, and then infused the genetically modified blood cells back into the patient’s
bloodstream

6. Gene therapy
Somatic gene
therapy
In - Vivo
Ex - Vivo
Germ line gene
therapy
In march 2006 an international group of scientists announced the successful use of gene
therapy to treat two adult patients for a disease affecting myeloid cells. The study is believed
to be the first to show that gene therapy can cure disease of the myeloid system. (by using
the concept of microRNA given by the team of scientists from the San Raffaele Telethon
Institute for Gene Therapy (HST-TIGET) in Milan (Italy).
The therapy strengthened Ashants’s immune system. She no longer had recurrent colds, was
allowed to attend the school. This procedure was not a cure. The white blood cells treated
genetically only work for few months, and the process needs be repeated every few month.

7. DIFFERENTIATION OF TYPES OF
GENE THERAPY
• Germline Gene Therapy • Somatic Gene Therapy
 In Germline Gene Therapy (GGT) germs
cells are modified by introduction of
therapeutic genes.
 Modifying a germ cell causes all the
organism cells to contains the modified
gene.
 The change is therefore, heritable and
passed on to later generation.
 For safety, ethical and technical reasons it
is not being attempted at present.
 In somatic cell gene therapy (SCGT) the
therapeutics genes are transferred into any
cell other than a germ cell.
 Introduction of genes into bone marrow
cells, blood cells and skin cells.
 It will not be inherited to later generation.
 Most focus on severe genetic disorder
including immuno-deficiencies
hemophilia, thalassemia and cystic
fibrosis.

8. TYPES OF SOMATIC CELL GENE
THERAPY

9. DESCRIPTION ON EX-VIVO AND IN-VIVO
 Isolate cells with defect from a patient.
 Grow the cells in culture.
 Introduction the therapeutic genes.
 Select genetically corrected cells and grow.
 Transplant the modified cells to the
patients.
 Direct delivery of therapeutic gene into target cells 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 (e.g.. Brain cells).
 In-vivo gene transfer is necessary when cultured cells
cannot be re-implanted in patients effectively.
• Ex-Vivo • In-vivo

10. Direct delivery
of therapeutic
cell into patient
body i.e., In Vivo
gene therapy
Representation of In-Vivo and Ex-Vivo gene therapy.
{HCC gene therapy is a treatment
method that uses gene products to
treat hepatocellular carcinoma
(HCC), also known as primary liver
cancer.}

11. VIRAL VECTORS IN GENE THERAPY AND
TYPES
Viral vector Non-viral vector
Viruses introduce their genetic material into the host cells
as a part of their replication cycle.
By removing the viral DNA and using the virus as a
vehicle to deliver the therapeutic DNA
• Retrovirus vector system: Recombinant retrovirus
have the ability to integrate into the host genome in a
stable fashion.
• Adeno Virus vector system: Useful in treating
common cold.
• Adeno Associated virus vector: It can integrate into
Chromosome 19.
• Herpes simplex virus vector: They persist into
nervous cell. These viruses have natural tendency to
infect particular type of cell
•The non-viral vector are naked DNA,
particle based and chemical based
•Direct introduction of pure DNA
construct into target tissues.
•Non-viral vectors are generally used
to transfer following type of nucleic
acids.
Small
DNA
Large
DNA
RNA

12. Physical Methods (Carrier Free Gene
Delivery)
•Needle injection: The genetic material is
administered through a needle carrying
syringe into tissue/ synthetic injection
from a vessel.
•Electroporation
•Gene Gun: Employs a high pressure
delivery system to shoot tissue with gold
or tungsten particle that are coated with
DNA.
•Hydroporation
Chemical Methods (Synthetic vector
based delivery)
•Inorganic particles: Ca. Phosphate,
Silica and Gold.
•Synthetic/natural biodegradable
polymer: Polyethyleneimine,
Polymethacrylate, Chitosan and
Dendrimers.
•Lipid Based: Lipid nano-emulsions,
solid lipid nano-particles and peptide
Non Viral
Vector
Physical
Method
Chemical
Method