NUCLEIC ACID BASED THERAPEUTIC DELIVERY SYSTEM by pramesh..pptx

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. NUCLEIC ACID BASED THERAPUTIC DELIVERY SYSTEM. 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 tha n 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. 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.

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

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