Gene Therapy: Breakthrough or Risk? Insights from Dr. Michiel Coppens

University courses Biological & Life Sciences
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Summary:
In a recent interview, Dr. Michiel Coppens shed light on the complexities of gene therapy, particularly for haemophilia A and B. While gene therapy promises a one-time solution for many genetic disorders, there are still many unknowns, including immune system reactions and differences in outcomes between haemophilia A and B patients. Dr. Coppens highlighted the challenges, such as the risk of liver complications and the mental strain of pre-screening, especially for patients with preexisting conditions like neutralising antibodies or liver damage. The interview emphasised the need for further research and caution in gene therapy applications.

Key Concepts:

  • Gene Therapy Basics: Explanation of gene therapy as a treatment approach, particularly for haemophilia A and B.
  • Immune System Complications: Around 80% of haemophilia A patients experienced liver enzyme (ALT) rises post-gene therapy, leading to corticosteroid treatment.
  • Immune Elimination: Presumption that the immune system may eliminate the therapy before it reaches the liver and delivers the therapeutic genes.
  • Screening and Mental Health: Emotional challenges for patients who undergo screening but may be disqualified due to neutralising antibodies or preexisting liver damage.
  • Knowledge Gaps: Emphasis on what remains unknown about long-term efficacy and safety of gene therapy, despite its potential.

As future scientists, what do you think are the ethical and psychological considerations we must address before widely implementing gene therapy?

Read the full interview here:

https://www.liebertpub.com/doi/10.1089/genbio.2024.29136.jmw

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This interview on gene therapy is fascinating! It’s clear there’s a lot of promise but also some serious challenges. What do you think are the biggest ethical and psychological issues we should tackle before gene therapy becomes more widespread?

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I’m glad you found the interview insightful! You’re absolutely right—while gene therapy holds tremendous promise, there are significant ethical and psychological considerations that need to be addressed. Ethically, one of the biggest issues is accessibility—who gets access to these potentially life-saving treatments, especially given the high costs? We also need to think about the long-term unknown risks, like possible complications down the line or unintended genetic changes.

Psychologically, the emotional toll on patients is considerable, especially with the pre-screening process. Imagine the mental strain of hoping for a treatment only to be disqualified because of something like neutralising antibodies or liver conditions. Additionally, even after treatment, the uncertainty of outcomes can create anxiety, as we still don’t know the full long-term effects of gene therapy. These issues need careful consideration before gene therapy can be widely adopted.

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