Today's lesson is focused on gene therapy, which we define as the introduction of genetic material into a patient to treat or prevent disease. Can anyone tell me why gene therapy is such an important aspect of modern medicine?

Exactly! Now, we have two main types of gene therapy: somatic and germline. Somatic gene therapy only affects the treated individual, while germline therapy can alter genes in egg or sperm cells. Let's remember this with the acronym **SAGE**: *Somatic and Germline Effects*.

Good question! Somatic therapy is widely accepted, while germline therapy is often controversial due to ethical concerns. Can anyone think of an example of somatic gene therapy?

Right! Well done. Let's summarize: Gene therapy can target a specific genetic defect in somatic cells without affecting future generations. Remember **SAGE** as a mnemonic for somatic and germline therapies.

In this session, we’ll cover gene delivery systems. Does anyone know the methods we use to deliver genes into cells?

Correct! Viral vectors, like AAV and lentiviruses, allow for long-term gene expression, while non-viral methods, like liposomes and electroporation, generally provoke a lower immune response. Let’s use the mnemonic **VINE** to remember: *Viral and Invasive Non-viral Expressing systems*.

Great question! The choice depends on disease type, required duration of expression, and safety. For instance, CAR-T therapy utilizes modified T-cells that are reinfused into the patient. Let's summarize: The delivery method is crucial in its effectiveness, and **VINE** helps us remember the two categories.

Let's discuss clinical applications. Can anyone cite a disease treated using gene therapy?

Exactly! Luxturna is an excellent example of successful gene therapy. Also, Zolgensma for Spinal Muscular Atrophy delivers the SMN1 gene. Remember **LZ**: *Luxturna for vision and Zolgensma for muscles*.

For cancer, we use CAR-T therapy, which engineered T-cells to target cancer cells. So, summarizing, gene therapy has real-world applications, and remember **LZ** to recall vision and muscle therapies.

We must also address the ethical aspects of gene therapy. Why do you think informed consent is crucial?

Great point! Informed consent ensures patients are aware of what they are undergoing. Additionally, germline editing is banned in various countries due to ethical concerns. Let’s remember **IRE**: *Informed consent, Regulation, Ethics*.

Regulatory bodies like the FDA in the USA, EMA in Europe, and ICMR in India. They oversee the safety of gene therapies. So, remember **IRE** for the essentials of ethical considerations in gene therapy.

Let’s discuss emerging trends in gene therapy. Can anyone name a new technology being researched?

Absolutely! CRISPR is revolutionary for gene editing and is being trialed for conditions like Sickle Cell Disease and Beta-Thalassemia. Let's remember **CGE**: *CRISPR, Gene Editing*.

Personalized gene therapies based on genome sequencing are paving the way for tailored treatments. Summarizing: Emerging trends like **CGE** highlight the future possibilities in gene therapy.