PPT-Gene Therapy In and Around the Germline

Kyle Orwig Magee Womens Research Institute University of Pittsburgh School of Medicine International Summit on Human Gene Editing A Global Discussion December 13 2015 Stem Cell Renewal. Of Which People, By Which People, For Which People?. Charis Thompson. Chancellor’s Professor . UC Berkeley / London School . of Economics. Washington, DC, December 2015. Consensus on Human . Germline. 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 . Ben Ho Park MD PhD. Johns Hopkins University. Financial Disclosures. I have financial relationships with commercial entities that are relevant to the content of this presentation.. Royalties from Horizon Discovery, LTD. 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 . 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 . We performed a genome-wide analysis of gene expressionto identify germline- and sex-regulated genes.Using mutants that cause defects in germ cell proliferationor gametogenesis, we identiÞed sets of g ICH Considerations General Principles to Address the Risk of Inadvertent Germline Integration of Gene Therapy Vectors TRANSMISSION TO CHMP November 2006 RELEASED FOR INFORMATION November 2006 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. (…and NINDS’ Adaptations). John D. Porter, Ph.D.. Program Director. National Institute of Neurological Disorders and Stroke. National Institutes of Health. MSG Conference: 9/22/2014. 2. Gene Therapy's Second Act. 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?.