Gene Therapy: Lesson 5 PDF

THE ASPECTS OF GENE THERAPY LESSON OBJECTIVES At the end of this topic, audience are expected to: describe gene therapy and it's forms; and assess the potential benefits and detrimental effects of gene therapy to global health. INTRODUCTION Gene Therapy is one of the modern attempts to...

THE ASPECTS OF GENE THERAPY LESSON OBJECTIVES At the end of this topic, audience are expected to: describe gene therapy and it's forms; and assess the potential benefits and detrimental effects of gene therapy to global health. INTRODUCTION Gene Therapy is one of the modern attempts to lessen the negative clinical consequences of hereditary illnesses. This developing subject has to be researched more by researchers and medical practitioners in order to fully realize its huge potential for eliminating worldwide pathologic conditions that are currently incurable. GENE SOMATIC GENE THERAPY: FORMS OF - Targets non-reproductive cells, affecting only the individual treated. GENE GERMLINE GENE THERAPY: THERAPY - Alters reproductive cells, potentially affecting future generations, though it's more controversial(arguable) and less practiced. EX VIVO GENE THERAPY: - Involves modifying cells outside the body and then reintroducing them. IN VIVO GENE THERAPY: - Directly targets cells inside the body with therapeutic genes.  BENEFITS OF GENE THERAPY 1. Treatment of Genetic Disorders: Gene therapy can target and correct the underlying genetic causes of inherited diseases like cystic fibrosis, muscular dystrophy, and certain types of cancer. 2. Personalized Medicine: It allows for personalized treatments tailored to an individual's specific genetic makeup, potentially increasing treatment efficacy and reducing side effects. 3. Potential for Curing Diseases: By addressing the root cause of genetic conditions, gene therapy holds the promise of curing diseases rather than just managing symptoms. 4. Advancements in Research: It drives innovation in genetic and molecular research, leading to new insights and therapies for a wide range of health issues. DETRIMENTAL EFFECTS OF GENE THERAPY 1. PeSafety Concerns: Gene therapy involves modifying genetic material, which can lead to unintended effects or off-target mutations, potentially causing new health issues or even cancer. 2. Ethical Issues: The ability to edit genes raises ethical concerns, such as the potential for "designer babies," where genetic modifications could be made for non-medical reasons. 3. Accessibility and Inequality: The high cost of gene therapy may limit access, potentially widening health disparities between wealthy and low- income populations. 4. Long-term Effects: The long-term effects of gene therapy are not fully understood, and there may be unforeseen consequences that only become apparent after extended periods.  SUMMARY In summary, while gene therapy offers revolutionary potential for treating and curing genetic disorders, it must be approached with careful consideration of its safety, ethical implications, and equitable access. 4o mini There are several approaches to gene therapy. These are the following: Replacement of mutated gene that causes disease with a healthy copy of the gene THE BASIC Inactivation of a mutated gene that is functioning improperly PROCESS Introducing a new gene into the body to help fight a disease VARIETY TYPES OF GENE THERAPY PRODUCTS There are a variety of types of gene therapy products, including (fda.gov 2018): Plasmid DNA is a key component of gene therapy and DNA vaccines, and is used to treat a variety of diseases including cancer, HIV-AIDS, Ebola, malaria, and influenza. Circular DNA molecules can be engineered genetically to bring therapeutic genes into human cells. Viral vectors are modified viruses that carry therapeutic genes into cells. Since viruses have a natural ability to deliver genetic material into cells, some gene therapy products are derived from viruses. Once viruses have been modified to remove their ability to cause infectious disease, these modified viruses can be used as vectors (vehicles) to carry therapeutic genes into human cells.  Bacterial vectors are modified bacteria that can be used to deliver DNA to cells and tissues for gene therapy. Bacteria can be modified genetically to prevent them from causing infectious disease and then used as vectors to carry therapeutic genes into human tissues. Human gene editing technology. Human gene editing technology The goal of gene editing is to disrupt harmful genes or to repair mutated genes Patient-derived cellular gene therapy products are a type of gene therapy that involves modifying a patient's cells to treat or cure disease. Patient-derived cellular gene therapy products Cells are removed from the patient, genetically modified and then returned to the patient.  GENE THERAPIES Gene therapy is a medical technique that involves modifying a person's genes to treat or prevent diseases. The process typically works by correcting defective genes, replacing faulty ones with healthy copies, or introducing new genes into the body to help fight diseases. Gene therapies can target both inherited genetic disorders, like cystic fibrosis or muscular dystrophy, and acquired diseases such as certain types of cancer. Gene Replacement - A defective gene is replaced with a functional one. Gene Editing - Techniques like CRISPR-Cas9 are used to directly modify or correct the defective parts of a gene. Gene Augmentation - New or modified genes are introduced to help the body fight diseases or compensate for abnormal genes. Gene Inhibition - Genes that cause disease are turned off or blocked. Gene therapy can be delivered in two main ways: In Vivo: Directly into the body. Ex Vivo: Cells are modified outside the body and then reintroduced. Gene therapies are potential therapies that can applied to a cure a wide range diseases, the purpose of this medical application is not only to reduce the symptoms of diseases but to eliminate the cuasative agent of the disease. Thank you!