Untitled Quiz

Gene Therapy Overview

• Gene therapy is a modern approach to treating hereditary illnesses.
• It aims to lessen negative consequences of hereditary conditions.
• Requires further research and medical practitioner involvement to realize full potential.
• Targets the elimination of currently incurable conditions worldwide.

Lesson Objectives

• Describe gene therapy and its types.
• Assess potential benefits and drawbacks of gene therapy on global health.

Forms of Gene Therapy

• Somatic gene therapy: Targets non-reproductive cells, affecting only the treated individual.
• Germline gene therapy: Alters reproductive cells, potentially affecting future generations. Less common due to controversial nature.
• Ex vivo gene therapy: Modifies cells outside the body and then reintroduces them.
• In vivo gene therapy: Directly targets cells inside the body with therapeutic genes.

Benefits of Gene Therapy

• Treatment of genetic disorders: Targets the root cause of inherited diseases like cystic fibrosis, muscular dystrophy, and some cancers.
• Personalized medicine: Tailored treatments based on an individual's genetic makeup, increasing efficacy and reducing side effects.
• Potential for curing diseases: Addresses the underlying genetic cause, potentially eliminating the disease rather than just managing symptoms.
• Advancements in research: Drives innovation in genetic and molecular research, leading to new insights and therapies for various health issues.

Detrimental Effects of Gene Therapy

• Safety concerns: Modifying genetic material has potential for unintended effects, off-target mutations, and new health issues, including cancer.
• Ethical issues: Gene editing raises concerns around "designer babies" and genetic modifications for non-medical purposes.
• Accessibility and inequality: High costs may limit access, exacerbating health disparities between wealthy and low-income populations.
• Long-term effects: The long-term consequences of gene therapy may not be fully understood until after extended periods.

Basic Process of Gene Therapy

• Replacement: Replacing a defective gene with a healthy copy.
• Inactivation: Inactivating a mutated gene that is malfunctioning.
• Introduction: Introducing a new gene to help fight a disease.

Variety of Gene Therapy Products

• Plasmid DNA: Key component of gene therapy and DNA vaccines, used to treat various diseases including cancer, HIV, Ebola, and malaria. Circular DNA molecules can be engineered to carry therapeutic genes.
• Viral vectors: Modified viruses that carry therapeutic genes into cells. Viruses naturally deliver genetic material, and then they are modified to not cause disease, before vectors carry therapeutic genes.
• Bacterial vectors: Modified bacteria used to deliver DNA to cells. Genetically modified to not cause infection.
• Human gene editing Technology: This technology aims to disrupt harmful or repair mutated genes.

Gene Therapies

• Gene therapy modifies a person's genes to treat or prevent diseases.
• This corrects defective genes, replacing faulty genes with healthy copies, or introduces new ones to fight diseases.
• Inherited genetic disorders (like cystic fibrosis/muscular dystrophy) and acquired diseases (e.g., cancer) can both be targeted..

Gene Therapy Techniques

• Gene Replacement: Replacing a defective gene with a functional one.
• Gene Editing: Using techniques like CRISPR-Cas9 to directly modify or correct parts of a gene.
• Gene Augmentation: Introducing new or modified genes to help the body fight disease or compensate for abnormal genes.
• Gene Inhibition: Turning off or blocking genes that cause disease.

Gene Therapy Delivery Methods

• In Vivo: Directly into the body.

• Ex Vivo: Cells are modified outside the body and then reintroduced.

• Gene therapies aim to cure a wide range of diseases by not just reducing symptoms but removing the cause.