PPT-Enzyme and gene therapy of enzyme defects

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. i BIRTH DEFECTS SURVEILLANCE: ATLAS ii i BIRTH DEFECTS SURVEILLANCEATLAS OF SELECTEDCONGENITAL ii Birth defects surveillance: atlas of selected congenital anomalies.Geneva: World Health Organization; FACTS. About 150,000 babies are born each year with birth defects. . The parents of one out of every 28 babies are told that their baby has a birth defect. There are over 4,000 known birth defects. Birth defects are the leading cause of death in the first year of life.. Hemodynamics, Pharmacology, and Updates. Amanda L. Affleck CRNA, MAE. Providence Anesthesia Services. Five Basic Questions. Is the patient acyanotic or cyanotic?. Is pulmonary arterial blood flow increased or not?. Monitoring . Program. (ABDMP). ABDMP. 3 Components:. Surveillance. Prevention. Referral to Services. Surveillance. Surveillance. Goals: Timely, Accurate, Complete Data. Bureau . of Public Health . Statistics. 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. Manoj K. Mohanty and Baojie Zhang. Department of Mining and Mineral Resources Engineering. Southern Illinois University at Carbondale. Introduction. Objective. Project Methodology. Data Compilation. Data Analysis. 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. Antiretroviral Pregnancy Registry. LM Mofenson, V . Vannappagari. , AE . Scheuerle. , B Baugh,. KP Beckerman, H . Betman. , N . Chakhtoura. , K Dominguez, A . Pikis. , NS . Santanello. 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.. Division of Birth Defects and Developmental Disabilities Understanding Congenital Heart Defects Congenital heart defects are conditions present at birth that aect the structure and function of t 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. (…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.