Gene Therapy in Cancer Treatment Presented by: Anirban Kumar Bora B.Pharm 4 th Year Roll: 19301913006 Supervisor: Mr. Kh. Hassan Reza
Introduction Main Cause Mutations in its DNA Reason Exposure to Carcinogens (e.g. tobacco) Exposure to Viruses Epigenetic factors (e.g. tumour promoter effects) Activation of Proto- oncogenes to Oncogenes Inactivation of Tumour Suppressor genes (e.g. BRCA1 & 2)
Gene Therapy - Introduction Use of nucleic acids for treatment, cure or prevention Replacement of a defective gene with its functional copy
Main Types Immunotherapy Oncolytic Virotherapy Gene Transfer
1. Immunotherapy Uses GM cells and viral particles to stimulate the immune system to destroy cancer cells; e.g. lung cancer.
Immunotherapy
2. Oncolytic Virotherapy Uses viral particles that replicate within the cancer cell to cause cell death; e.g. metastatic cancers.
3. 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.
Some Therapeutic Approaches
by introducing specificity into T cells Delivering CAR Recognize antigen of choice on cancer cells Facilitate tumour cell recognition Formation of activated T cells Killing of Target cells 1. B-cell leukaemia and lymphoma: Immunotherapy
2. Head and neck squamous cell carcinoma: By restoration of functional p53 using a recombinant adenovirus expressing under a Rous sarcoma virus promoter. This is the first commercialized gene therapy, named Gendicine TM . By in vivo intratumoural injection. Gene Transfer
3. Malignant glioma: HSV-TKase (vector NV1020) has been used to convert the prodrug Ganciclovir into cytotoxic triphosphate ganciclovir. Commercial example: Cerepro (cytomegalovirus promoter). Oncolytic Virotherapy
Cancer Type Commercial Name Approach Type of Therapy Prostate Cancer Prostvac TRICOM vector vaccine Immunotherapy Soft Tissue Sarcoma TNFerade Adenoviral vector Gene Transfer Pancreatic Cancer Rexin-G Retroviral vector Gene Transfer Other examples
Conclusion Serenities iPSC technology Hope for orphan and other diseases Concerns Translation of iPSC technology into the clinics AAV immunogenicity
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