PPT-Gene Therapy for Fanconi Anemia: A Primer

1 Fanconi Anemia What is Fanconi Anemia FA 2 DNA damage Eventual cell death FA Cell Healthy Cell DNA damage Production of more healthy cells Repairs itself When this occurs in bone marrow BM cells for example it leads to BMF or leukemia. Anthony Winchell. What is Fanconi Anemia?. http://www.sitemaker.umich.edu/nhowlett/files/fa-qr.gif. What is the Fanconi Anemia pathway?. http://www.hindawi.com/journals/anemia/2012/926787/. What is Fanconi Anemia Complementation Group L?. 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. CKD . Patients. 2012 KDIGO . Update. Dr. . Shahram. . Taheri. . CKD is a worldwide public health issue. . The incidence and prevalence of kidney failure are increasing, outcomes are poor, and the cost is high. . 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 . Study . Knock-Out Mutations. ?. Elaine Chiu. Nancy Phang. June 4, 2009. Isolation. Grow plants.. Collect leaves.. Isolate DNA from the leaves.. How do we screen for . T-DNA inserts?. FW. 5. ’. 5. ’. This kind of study was conducted �rst time in Pakistan. Its objective was to ascertain the associated clinical features and analyze the FANCA exon 28 and exon 29 mutations in Pakistani Fanc 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. Though internists frequently encounter iron deficiency anemia, we must remain vigilant to accurately diagnose more complex cases.. LABORATORY EVALUATION. BONE MARROW EXAM. CONCLUSIONS. The patient was diagnosed with . MD. ASSTT. PROFESSOR. DEPTT. OF MEDICINE. Aplastic. anemia is . pancytopenia. with bone marrow . hypocellularity. .. M. en and women are affected with equal frequency.. A. ge distribution is biphasic, with the major peak in the teens and twenties and a second rise in older adults.. 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?.