PPT-Gene Therapy and Viral Vector

Lecture 1 and 2 Gene Therapy Definition the introduction of normal genes into cells in place of missing or defective ones in order to correct genetic disorders Approaches Replacing a mutated gene that causes disease with a healthy copy of the gene. Will Herrick. Peyton Group Meeting. March 20, 2013. What Are Adenoviruses?. Linear . dsDNA. virus. 80-100 nm. Nonenveloped. Icosahedral structure:. 1: penton . capsomeres. 2: . hexon. . capsomeres. . Genica. . Prof. . Saggio. Tutor Dott.ssa . Piersanti. Carolin . Tauber. Graziana. . Luciotto. Ludovica. . Taglieri. Veronica . Cacciamani. Bianca . Fabi. Gene . Therapy. . for. Parkinson . Disease. . Gene therapy: . to correct a . genetic defect . by transferring of a . functional normal copy. of the gene into cells. Examples of diseases caused by genetic defect. Ornithine . transcarbamylase. to correct a . genetic defect . by transferring of a . functional normal copy. of the gene into cells. Examples of diseases caused by genetic defect. Ornithine . transcarbamylase. (OTC deficiency). 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.. where . are we now and . where . are we going? . Jintanat. Ananworanich, MD, PhD. Associate Director for Therapeutics Research. US Military HIV Research Program (MHRP). Maryland, USA. Deputy Director of SEARCH . Will Herrick. Peyton Group Meeting. March 20, 2013. What Are Adenoviruses?. Linear . dsDNA. virus. 80-100 nm. Nonenveloped. Icosahedral structure:. 1: penton . capsomeres. 2: . hexon. . capsomeres. 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. 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 . Cell therapy is defined as the administration of live whole cells or maturation of a specific cell population in a patient for the treatment of a disease. . – Bone Marrow Transplantation. Bone . Marrow Transplantation for DBA. Cary O. Harding, MD. Department of Molecular & Medical Genetics. Disclosures. BioMarin Corporation. Funds for participation in clinical trials. Sapropterin dihydrochloride. rAvPAL-PEG. National PKU Alliance. Rubanraj. . Dept of Mathematics. St. Joseph’s College. Trichy. Vectors . Vector is an agent that can carry a DNA fragment into a host cell in which it is capable of replication. If it is used only for reproducing the DNA fragment, it is called a . 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..