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. How they are caused and why they can be beneficial. What kind of genetic disease?. Can be of two kinds:. Caused by . environmental factors. Hereditary. Genetic disease by environmental factors includes cancer due to the cells undergoing . INTRODUCTION. Most developments in biotechnology originated for their potential applications in health care.. Contribution of biotechnology:. Diagnosis of diseases. Therapeutic agents. Correction of genetic disease-Gene therapy. 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. By Kole Drumheller and Austin Dillard. Gene Therapy. What is Gene therapy?. This is a technique that is designed to introduce new genetic material into cells to help fix abnormal genes or to make a new protein that will help the person.. Chapter 19, lesson 3 . objective:predict. the consequences of refusal skills in various situations. Words to know. Heredity: the passing of traits from parents to their children. chromosomes: threadlike structures found within the nucleus. Outline. Genetic Diseases Background. Recessive Genetic Diseases. Dominant Genetic Diseases. Pedigrees. What is a . G. enetic . D. isease?. Definition: A . genetic disease . is a disease that is inherited from one’s parents and is present from the moment the zygote is created.. 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.. Mohanad. Mahdi Al-. Hindawi. . Introduction . A . gene. (from ancient Greek: . gonos. : . offspring. , procreation) is a locus (or region) of DNA which is made up of nucleotides.. The transmission of genes to an organism's offspring is the basis of the inheritance of phenotypic traits. . 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. Gene therapy . is when DNA is introduced into a patient to treat a genetic disease. The new DNA usually contains a functioning gene to correct the effects of a disease-causing mutation. . Gene therapy uses sections of DNA (usually genes) to treat or prevent disease. The DNA is carefully selected to correct the effect of a mutated gene that is causing disease. The technique was first developed in 1972 but has, so far, had limited success in treating human diseases. Gene therapy may be a promising treatment option for some genetic diseases, including . 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. Dr Anke . Hensiek. , Dr med, PhD, FRCP. Affiliated Assistant Professor University of Cambridge. Consultant Neurologist. Genetherapy. . Correct and precise correction of the DNA defect directly. Compensate a disease mechanism. 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..