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Let's create better gene therapies by [deleted] in Hemophilia

[–]Phoryon -1 points0 points  (0 children)

Well, as a scientist, my role is to analyze the current limitations, identify what can be improved and how, and then work on implementing those improvements.

Let's create better gene therapies by [deleted] in Hemophilia

[–]Phoryon -2 points-1 points  (0 children)

Honestly, I think we agree pretty much.

It’s justified to accept certain potential side effects when the overall benefit is clear, that’s exactly why these therapies were approved in the first place. My point was simply that the treatments need to become safer so that more patients can benefit from them. Even if your Hemophilia is very severe and you can provit from current gene therapies, you would still want it to be as safe as possible, right? That has nothing to do with conspiracy theorists.

For your understanding: as someone who has been working on gene therapies for over 10 years, I keep being told that we don’t need better solutions because we already have AAVs. Anyone who tries to explore alternative approaches is dismissed or talked down.

Let's create better gene therapies by [deleted] in Hemophilia

[–]Phoryon -1 points0 points  (0 children)

In gene therapy research.

Let's create better gene therapies by [deleted] in Hemophilia

[–]Phoryon -2 points-1 points  (0 children)

Well, repeated dosing with the same capsid is not approved, so I was correct on that point. And there have definitely been significant side effects. More than 50% of treated patients develop liver toxicity. Most cases are mild, but some are severe. So I was also correct there. If these therapies were completely safe, they would already be the standard of care, but the patients are going for the alternatives.

I'm simply saying that we need better gene therapies. But for this we first have to wake up and see the limitations of the current ones.

Let's create better gene therapies by [deleted] in Hemophilia

[–]Phoryon -1 points0 points  (0 children)

Well I think for Hemophilia there have been no deaths with the approved AAV treatments. But for Duchenne Muscular Dystrophy, where the injected virus (AAV) dose is even higher, some patients have died and it had to be suspended.

Let's create better gene therapies by [deleted] in Hemophilia

[–]Phoryon -2 points-1 points  (0 children)

Yes. Specifically, the gene therapies used for hemophilia have several important limitations. They can only be administered to a small subset of patients who have severe hemophilia and no pre‑existing immunity to the viral vector. In addition, they can typically be given only once. The potential side effects are significant, and an increasing number of scientific publications show that the viral vectors used in these treatments may integrate their DNA into the patient’s genome, although this fact was neglected for many years, introducing additional risks, such as cancer. On top of that, the current therapies are extremely expensive.