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Emerging Treatments, Complex Disease and Pharmacogenetics and Brittle Bones (PART 3/5) PassPom 2025: Crashcourse Day 3

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Summary

Gain an in-depth understanding of protein related therapy, specifically focusing on its mutation specificity, in this engaging on-demand teaching session. The course covers how chaperones aid in correcting misfolded proteins, an essential process in treating Fabry's disease. The discussion extends to the role of modulators, particularly Ivacaftor, in treating cystic fibrosis by activating the inactive chlorine ion channel to reduce mucus viscosity. We assure you this knowledge could be a great add-on to your medical expertise.

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Description

🚨 Calling all first-year medics! 🚨

Struggling with POM? Want to ace your exams with expert-led teaching? πŸ©ΊπŸ“š Join our POM Crash Course – a FREE, interactive lecture series designed to break down key concepts and boost your confidence! πŸ’‘βœ¨

πŸ“… Date: 3 / 04 / 2025

πŸ“ Location: Medall

Learning objectives

  1. Understand the role of chaperones in protein folding and their relevance in genetic disorders.
  2. Comprehend the implications of misfolded proteins and how they can affect the functioning of the body.
  3. Understand how chaperones can help with specific diseases like Fabry's disease, and how the treatment is mutation-specific.
  4. Grasp the function of modulators in diseases like cystic fibrosis, especially how they can activate or deactivate certain processes, with a focus on the specific example of Ivacaftor.
  5. Interpret the impact of modulators on the body's functions and their role in making bodily substances, such as mucus, less viscous, thereby improving the body's functioning.
Generated by MedBot

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Computer generated transcript

Warning!
The following transcript was generated automatically from the content and has not been checked or corrected manually.

Hello. Uh on my back. Hi there. Ok. Where was I? Was I at Chaperones or modulators? Hello. Perfect. Ok. Chaperones, er, protein folding fails uh misfolded proteins. Megalota helps with fabrys disease. I've said this about three times and I took myself, which is annoying but basically, uh a big thing about uh charo and any sort of protein related therapy, they're mutation specific if you don't take away anything. Remember that uh only works when you have the right mutation. Uh and basically make sure that your misfolded protein is folded into the correct shape and therefore can work. Uh modulators activate or deactivate uh in cystic fibrosis, you've got an inactive chlorine ion channel. Uh And Ivacaftor remember that, please, we open that channel allowing for the chlorine iron to get into the mucus. Uh making sure it's not as viscous. Very simple uh agonist and antagonist. I hope you guys understand that.