Ep 50: Changing Lives by the Million - CRISPR Cures

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We Love Science podcast celebrates our 50th episode with a discussion all about the hopes and progress of CRISPR clinical trials. Before a new drug, therapy, vaccine, or medical device is approved for general use by the public, its safety and effectiveness are evaluated in a series of tests on volunteers. These Clinical Trials progress in 3 successive phases with each phase designed to answer specific questions about the product under evaluation. Questions can include: What side effects does it cause? What is the lowest effective dose? What is the highest safe dose? Does it improve patients’ quality of life? Is it an effective cure for the disease in question? How does it compare to the current treatments (if any are available) for that particular disease?

Currently, only one CRISPR gene therapy, Casgevy, is approved for public use. It is approved in several countries to treat the blood disorders, Sickle Cell Disease and Beta Thalassemia. However, many CRISPR clinical trials are still ongoing; they span ~ 10 major disease categories and occupy every clinical trial phase. In this episode, we discuss the successes and challenges of CRISPR clinical trials to cure HIV/AIDS, Cardiovascular disease, and others.

Tune in to learn more about:

· What questions do successive clinical trial phases answer?

· How can CRISPR technology cure HIV/AIDS?

· The results for the hereditary amyloidosis treatment, which is now in phase III of clinical trials

· Listener question: An update for Sickle Cell Disease CRISPR treatments
For more information on this topic, visit our website: welovesciencepodcast.com

Useful Links: US and EU clinical trials databases:

https://clinicaltrials.gov/

https://euclinicaltrials.eu/?lang=en
Jump directly to the next episodes in the CRISPR series:

• Ep 53: Safety and Ethics in a CRISPR world

Catch up on the previous CRISPR episodes:

• Ep 35: The first CRISPR Chronicles episode: How CRISPR gene therapy is bringing hope to the world
• Ep 38: A foundational discussion explaining the secret behind CRISPR gene therapy
• Ep 41: A look into the ups and downs of how the CRISPR saga developed
• Ep 47: The CRISPR Cure for Sickl

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Music from Pixabay: Future Artificial Intelligence Technology 130 by TimMoor
Music from https://freemusicarchive.org/music/Scott_Holmes: Hotshot by ScottHolmesMusic

Chapitres

1. Ep 50: Changing Lives by the Million - CRISPR Cures (00:00:00)

2. How do clinical trials work? (00:03:47)

3. Strategy for CRISPR-mediated HIV cure (00:10:03)

4. Progress of clinical trial for HIV cure (00:13:51)

5. Challenges faced by the HIV clinical trial (00:17:22)

6. Overview of clinical trials for lupus and cardiovascular disease (00:20:09)

7. How CRISPR is being used to augment other biotechnology (00:22:59)

8. Overview of the phase 3 clinical trial for hereditary ATTR disease (00:24:04)

9. Strategy for treating hATTR with CRISPR gene therapy (00:26:52)

10. Results from the hATTR clinical trial (00:30:19)

11. Update for the Sickle Cell Disease CRISPR cure, Casgevy (00:34:35)