PPT-Safety Evaluation of IV-administered BBP-812, an AAV9-based Gene Therapy for the Potential

May 13 2021 David Scott 2 I am a shareholder and employee of BridgeBio Pharma Inc the parent company of Aspa Therapeutics 3 4 Canavan disease is characterized by a loss of Aspa expression and a systemic build up of Nacetylaspariticacid NAA. 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.. 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 . 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. 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?. 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 . 2014;18:151–161; 2. Ginn SL, et al. . J Gene Med.