PPT-Genetic Diseases and Gene Therapy Approaches

2020 Monogenic and Complex Diseases Gene therapy is a promising approach for both monogenic and complex diseases 1 1 Wang D Gau G Discov Med 201418151161 2 Ginn SL et al J Gene Med. Risks and Benefits of Knowing Your Genetic Makeup. Julie Hopp. Genetic Screening: Who Should Be Tested. CDC’s 2004 Science Ambassador Program. Overview. Genetics Review. Types of Genetic Disorders. William Brooks. Medicinal Chemistry. March 31, 2011. Outline. Conventional drug design vs. gene therapy. Examples of genetic engineering. Types of gene therapy. Mechanisms. Delivery vectors. Viral. Non-viral. Kyle Orwig. Magee-. Womens. Research Institute. University of Pittsburgh School of Medicine. International Summit on Human Gene Editing:. A Global Discussion. December 1-3, 2015. Stem Cell. Renewal. Therapy of enzyme defects: general considerations. How many organs are affected by the enzyme defect: One organ, a few, or all organs?. How severe is the defect?. Can the defect be adequately controlled by conventional treatment?. Genetic Counseling. Treating . Genetic . Disease. Removing . an affected body . part.. Replacing . an affected body part or biochemical . with material . from a . donor.. Delivering . pure, human proteins derived . Polydactyly. Down Syndrome. Hemophelia. Progeria. Hypertrichosis. Sickle Cell Anaemia. INTRODUCTION. What is genetic diseases/disorders?. The human body is composed up of cells , each one specializing a particular function like sensing light, smelling . Dept. of Medical Genetics & Medical Biology. Gene Therapy cartoon 10 - search ID shrn157. . . Gene Therapy cartoon 5 - search ID shrn147. . . Purpose of. gene therapy:. Management and correction of human diseases. 6000. inherited human diseases catalogued to date, only a few are currently treatable.. Gene therapy. —introducing functional copies of a gene into an individual with two defective copies of the gene—is a potential tool for treating inherited human diseases.. GENETIC. ENVIRONMENTAL. BOTH. CONGENITAL. HEREDITARY. FAMILAL. MUTATIONS. PERMANENT change in DNA. GENE. MUTATION: (may, and often, result in a single base error). CHROMOSOME . MUTATION: (visible chromosome change)part of chromosome. December 1984 NTIS order #PB85-206076 “Where two principles really do meet which cannot be reconciled with one another, theneach man declares the other a fool and heretic. ” –Ludwig Wit Msc. . . level. College of Pharmacy . Al-. Mustansiriyah. University. Dr. . Basma. Al- . Sudani. Lecture . 2. 9-11-2021. Table. . of Content. What Genes can do. Why Genetic Disorders. Law of Inheritance. ?. G. enetic . engineering. , the artificial manipulation, modification, and recombination of DNA or other nucleic acid molecules in order to modify an organism or population of organisms. . This . may mean changing one base . techniques . Content. Introduction. Importance of Gene transfer. Types of gene transfer techniques. Method. Application. Introduction. Gene transfer is to transfer a gene from one DNA molecule to another DNA molecule.. Gene therapy . Genetic Engineering. Use the website to research the answers to the questions on your sheet.. Be ready to discuss the answers next lesson.. Using technology to modify organisms. Recombinant DNA technology can now be used to alter the genetic make-up of organisms much more quickly..