Gene therapy

Описание: Join for an engaging lecture designed for bachelor's biology students exploring the revolutionary field of Gene Therapy. This isn't just treating symptoms—it's fixing the fundamental genetic cause of disease.

We dive deep into the science behind using DNA and RNA as medicine. We'll break down the critical challenge of delivery, comparing and contrasting the highly efficient world of Viral Vectors (like AAV and Lentivirus) with the safety-focused advancements in Non-Viral Vectors (like Lipid Nanoparticles).

Understand the therapeutic goals—from corrective gene replacement to gene silencing—and tackle the major hurdles facing the field. We'll specifically analyze critical safety concerns, including the immune response, off-target effects, and the risk of insertional mutagenesis. Finally, we'll discuss the ethical and financial challenges of bringing million-dollar cures to the people who need them.

Key Topics Covered:

Therapeutic Payloads: Corrective genes, siRNA, and suicide genes.
Delivery Systems: In Vivo vs. Ex Vivo methods.
Viral Vectors: AAV, Lentivirus, Adenovirus.
Safety Concerns: Immunogenicity and Off-Target Effects.
Future Outlook: CRISPR and personalized medicine.