LESSON 12 GENE THERAPY

Summary

This educational video explains the fundamentals of gene therapy, focusing on how genes are used to treat or prevent diseases. It covers the role of stem cells, including embryonic, adult, induced pluripotent, and umbilical cord stem cells, in gene therapy. The video explores the different types of stem cells, their potential benefits, and the ethical considerations surrounding their use. The viewer will gain an understanding of stem cell applications, including their use in regenerative medicine and cancer treatment. The video emphasizes the promising future of stem cells in revolutionizing healthcare and disease treatment.

Takeaways

• 😀 Gene therapy is a technique that uses genes to treat or prevent diseases by inserting normal genes into cells to replace abnormal ones.
• 😀 Various approaches in gene therapy include replacing altered genes, inactivating mutated genes, and introducing new genes to fight diseases.
• 😀 Stem cells play a crucial role in gene therapy due to their ability to self-renew and differentiate into specialized cells.
• 😀 Stem cells are classified by potency: totipotent (can form all cell types), pluripotent (can form many but not all cell types), and multipotent (can form a limited set of cell types).
• 😀 Embryonic stem cells are pluripotent, capable of forming any cell in the body, but their use raises ethical concerns and risks such as immune rejection and teratoma formation.
• 😀 Adult stem cells are multipotent, meaning they can become various specialized cell types but are more limited than embryonic stem cells.
• 😀 Induced pluripotent stem cells (iPSCs) are adult cells reprogrammed to behave like embryonic stem cells and can be used in treating diseases like Parkinson's or Alzheimer's.
• 😀 Umbilical cord stem cells are readily available, inexpensive, and multipotent, and can be stored for future use.
• 😀 Despite the promise of stem cells in gene therapy, ethical controversies surrounding the sourcing and use of these cells remain a significant challenge.
• 😀 Stem cell therapies, like hematopoietic stem cell transplantation, are already being used to treat blood cancers like leukemia, with the potential to revolutionize medicine.
• 😀 The ongoing research and development in stem cell technologies are expected to lead to groundbreaking treatments and a potential new medical revolution, much like the industrial revolution did for technology.

Q & A

What is gene therapy and how does it work?

Gene therapy is a technique that uses genes to treat or prevent diseases. It involves inserting healthy genes into a person's cells to replace or repair defective genes that cause diseases. The healthy gene may be delivered using vectors, such as viruses, to transport it into the cells.

What are the main approaches used in gene therapy?

The main approaches in gene therapy include: 1) Replacing an altered gene that causes disease with a healthy copy. 2) Inactivating or knocking out a mutated gene that functions improperly. 3) Introducing a new gene into the body to help fight a disease.

Why are stem cells important in gene therapy?

Stem cells are important in gene therapy because they have the unique ability to self-renew (make copies of themselves) and differentiate into various types of specialized cells, such as blood, nerve, or muscle cells. This makes stem cells crucial for replacing damaged or lost cells in the body.

What is the difference between self-renewal and differentiation in stem cells?

Self-renewal refers to a stem cell's ability to divide and produce identical copies of itself, ensuring a constant supply of stem cells. Differentiation is the process where a stem cell matures into a specialized cell type, like a muscle or blood cell, which performs specific functions in the body.

What does the term 'potency' refer to in stem cell biology?

Potency refers to a stem cell's ability to differentiate into various types of specialized cells. Totipotent stem cells can become any cell type in the body and even contribute to the formation of the placenta. Pluripotent stem cells can become any cell type in the body, but not the placenta. Multipotent stem cells can only become a limited number of cell types.

What are the different types of stem cells used in gene therapy?

The main types of stem cells used in gene therapy are: 1) Embryonic stem cells (ESCs), which are pluripotent and can become any cell type in the body. 2) Adult stem cells (ASCs), which are multipotent and can only become a limited number of cell types. 3) Induced pluripotent stem cells (iPSCs), which are adult cells reprogrammed to behave like pluripotent stem cells. 4) Umbilical cord stem cells, which are multipotent and can form many different cell types.

What are the advantages of using embryonic stem cells in gene therapy?

Embryonic stem cells are advantageous because they are pluripotent, meaning they can differentiate into any cell type in the body. They also have the potential to self-renew indefinitely, which allows for the creation of large quantities of cells for therapeutic use.

What are the ethical concerns related to the use of embryonic stem cells?

Ethical concerns regarding embryonic stem cells mainly focus on how they are obtained, as they are derived from human embryos. The process of harvesting these cells raises questions about the moral status of embryos and the potential for exploiting embryos in research.

How are induced pluripotent stem cells (iPSCs) created and what are their potential benefits?

Induced pluripotent stem cells (iPSCs) are created by reprogramming specialized adult cells, like skin cells, to behave like embryonic stem cells. This reprogramming is achieved by introducing specific genes into the adult cells. iPSCs are beneficial because they can become any cell type in the body, offering a potential method for treating various diseases like Parkinson's or Alzheimer's without the ethical issues associated with embryonic stem cells.

What is the process of hematopoietic stem cell transplantation in treating leukemia?

Hematopoietic stem cell transplantation involves taking healthy blood-forming stem cells from the patient or a donor (often from umbilical cord blood) and transplanting them into a patient with leukemia. The patient's abnormal blood cells are destroyed using chemotherapy or radiation, and the transplanted stem cells help regenerate healthy blood cells, allowing the patient to fight infections and recover.