PPT-Advances in gene therapy for phenylketonuria (PKU)

Cary O Harding MD Department of Molecular amp Medical Genetics Disclosures BioMarin Corporation Funds for participation in clinical trials Sapropterin dihydrochloride rAvPALPEG National PKU Alliance. RESEARCH ADVANCES www.FightBlindness.org Winter 2013 Landmark gene therapy restores vision in children and young adults More than 40 children and young adults who were virtually blind have had some RESEARCH ADVANCES www.FightBlindness.org SUMMER 2014 Landmark Gene Therapy Restores Vision in Children and Young Adults More than 40 children and young adults who were virtually blind have had som Biologia Celular e Molecular II. 2012/2013. Work done by:. Cátia Ferreira (T5). Isa Costa (T6) . Jéssica Vasconcelos (T5). Sara Ferreira (T6). Cellular and Molecular . M. echanisms in Phenylketonuria. 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. Genetic Engineering. Technology that involves modifying the DNA of one organism by inserting the DNA of another.. First used in the . 1970s. Today is used in . a wide variety of . applications. Uses of Genetic Engineering. 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 Engineering. Technology that involves modifying the DNA of one organism by inserting the DNA of another.. First used in the . 1970s. Today is used in . a wide variety of . applications. Uses of Genetic Engineering. 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 . 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?.