Important: The BOOK: Gene Therapy (Special Topics in Biology Series) (Paperback) by Michael A. Palladino (Author), Mary Colavito (Author)
It costs $10 only. Required. Course will be graded according to two exams (50 ppt for each of them).
Lecture 1 General principles of Gene Therapy. Diseases that could be treated by gene therapy. Clinical trials. Gene Delivery. Reporter genes and Transfer efficiency. Germline vs. somatic cells. In vivo and ex vivo gene therapy. Ex vivo and In vivo Gene Therapy.
Lecture 2. DNA–based gene therapy. Mechanical methods of delivery. Example: Duchenne myotrophy. Liposomal methods of delivery. Cystic fibrosis.
Lecture 3 Retroviral and Adenoviral vectors. Safety of GT vectors. Gutted vectors. Helper viruses and Packaging cell lines. Examples: ornithine transcarbamylase deficiency (OTC). Severe Combined Immunodeficiency (SCID). Failures in the OTC and SCID GT trials.
Lecture 4. Adeno-associated virus- based vectors. Hemophilia. Diabetes mellitus. Erythropoietin. How to target vector to the particular cell. Tissue specific promoters. Vectors as ligands for cellular receptors.
Lecture 5. Сancer gene therapy. Genetic Prodrug Activation Gene Therapy. GT methods to overcome chemotherapy resistance of tumors. Biological response modifiers. Oncolytic virus- based strategies of tumor treatment. DNA vaccines for cancer and infectious diseases. AIDS as a target for DNA vaccines. Antisense based methods of gene therapy.