POTENTIALTARGETDISEASESFORGENETHERAPY (1).pptx

Hello here i upload a pptx for you about disease.

1. POTENTIAL TARGET DISEASES FOR GENE THERAPY Presented by: Neha kannojiya M.Pharm . (Pharmaceutics) 2 nd sem. Faculty of Pharmacy Lucknow

2. What is a Gene ? It is a segment of Deoxyriboneuclic acid(DNA) needed to contribute to a function. Genes are composed of DNA, a molecule in the memorable shape of a double helix. A gene is a sequence (a string) of bases. It is made up of combinations of adenine (A), thymine (T), cytosine (C), and guanine (G)

3. What is Gene Therapy ? The term gene therapy describes any procedure intended to treat or alleviate disease by genetically modifying the cells of a patient. It encompasses many different strategies and the material transferred into patient cells may be Genes Gene segments Oligonucleotides.

4. Potential target diseases for Gene Therapy Three types of diseases prospected for gene therapy can be distinguished Monogenic disorders , single locus (gene) is defective 100% heritable. Examples: Sickle cell anemia , Severe Combined Immunodeficiency (ADA-SCID / X-SCID), Cystic fibrosis, Hemophilia , Duchenne Muscular dystrophy, Huntington’s disease, Parkinson’s Polygenic disorders , multiple genes involved. Examples: Heart disease, Cancer, Diabetes, Schizophrenia and Alzheimer’s disease. Infectious diseases , such as HIV.

5. Inherited Disorders Severe Combined Immune Deficiency (ADA-SCID)  ADA-SCID is also known as the bubble boy disease. Affected children are born without an effective immune system and will succumb to infections outside of the bubble without bone marrow transplantation from matched donors. The therapeutic gene called ADA was introduced into the bone marrow cells of such patients in the laboratory, followed by transplantation of the genetically corrected cells back to the same patients. The immune system was reconstituted in all six treated patients without noticeable side effects, who now live normal lives with their families without the need for further treatment. 

6. 2. Chronic Granulomatus Disorder (CGD) CGD is a genetic disease in the immune system that leads to the patients inability to fight off bacterial and fungal infections that can be fatal. Using similar technologies as in the ADA-SCID trial, investigators in Germany treated two patients with this disease, whose reconstituted immune systems have since been able to provide them with full protection against microbial infections for at least two years.

7. 3. Hemophilia Patients born with Hemophilia are not able to induce blood clots and suffer from external and internal bleeding that can be life threatening. In a clinical trial conducted in the United States , the therapeutic gene was introduced into the liver of patients, who then acquired the ability to have normal blood clotting time. The therapeutic effect however, was transient because the genetically corrected liver cells were recognized as foreign and rejected by the healthy immune system in the patients. This is the same problem faced by patients after organ transplantation, and curative outcome by gene therapy might be achievable with immune-suppression or alternative gene delivery strategies currently being tested in preclinical animal models of this disease.

8. 4. Blindness Leber's Congenital Amaurosis (LCA) is a rare inherited eye disease that appears at birth or in the first few months of life Researchers at Moorfields Eye Hospital and University College London in London conducted the first gene therapy clinical trial for patients with RPE65 LCA. The first patient was operated upon in early 2007. Researchers at Children's Hospital of Philadelphia and the University of Pennsylvania have treated six young people via gene therapy. Eye Surgeon Dr. Al Maguire and gene therapy expert Dr. Jean Bennett developed the technique used by the Children's Hospital

9. Cancer Gene Therapy Multiple gene therapy strategies have been developed to treat a wide variety of cancers, including Suicide gene therapy, Oncolytic virotherapy , Anti-angiogenesis and Therapeutic gene vaccines.

10. Types of Gene Therapy used against Cancers. Immunotherapy Oncolytic Virotherapy Gene Transfer

11. 1. Gene Transfer Introduces new genes into a cancerous cell or the surrounding tissue to cause cell death or slow the growth of cancer e.g. in solid tumours . 2. Immunotherapy Uses Genetically manipulated cells and viral particles to stimulate the immune system to destroy cancer cells e.g. lung cancer. 3. Oncolytic Virotherapy Uses viral particles that replicate within the cancer cell to cause cell death; e.g. metastatic cancers

12. Thank You