PPT-Outcomes: MDA-NINDS Workshop on Best Practices for Gene Therapy Programs (April 2014)

and NINDS Adaptations John D Porter PhD Program Director National Institute of Neurological Disorders and Stroke National Institutes of Health MSG Conference 9222014 2 Gene Therapys Second Act. Color Blindness. Discovery. John Dalton, an English chemist, was the first to report the disorder. . . . F. irst . general condition of color blindness was called . Daltonism. . Dalton believed that a colored liquid inside the . 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.. 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 . 187. Hg. Shamsuzzoha. . Basunia. Lawrence Berkeley National Laboratory. Berkeley, CA 94720. Specialized Evaluators Workshop. IAEA, Vienna, April 27-29, 2015. Motivation and Outline. Sharing . Difficulties/experiences. 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.. 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 . 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. Joanna Vivalda. Claudia Moy. Carlos Faraco . Peter Gilbert. Clint Wright. Coming together is a beginning.. Keeping together is progress.. Working together is success. . . ~Henry Ford. NIH . 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. Peter Marks, MD, PhD. National Press Foundation. November 17, 2023. Outline. Status of gene therapy and genome editing. Regulatory considerations for genome editing. Regulatory guidance for genome editing. Starter: Comparing DNA processes. Process. What does it do?. How does it work?. PCR (Polymerase Chain. Reaction). DNA Sequencing. DNA Profiling. Gel Electrophoresis. Process. What does it do?. How does it work?. coreHEM. : A core outcome set for gene therapy in hemophilia. Elizabeth Clearfield, MHS | May 8, 2023 . Polling Question. Do you believe a core outcome set adds value to the understanding of patient-important outcomes?.