Haemophilia A trial results 'mind-blowing'

Post by seren » Thu, Dec 14, 2017, 4:57 PM UTC

Chriky wrote: post #19

seren wrote: post #12

Earlier quoted context omitted.

> I think if this worked as intended, no additional treatment is required. It is correct if the therapy is targeting all stem cells. I have not understood clearly from the article but it seems the treatement is targeting "living cells". Once all your 'fixed' cells have been renewed you would need another round of treatement.

I have Haemophilia A, I was actually offered a place on an earlier trial although I didn't do it. It was explained to me that you would not need further gene treatments even though it targeted adult cells. I think most cells are made by mitosis and don't come from stem cells. Here's a screenshot of the explanation I received when I was invited on the trial - https://i.imgur.com/H329Vgv.jpg > "By using BMN 270 to prov…

Post by jessriedel » Thu, Dec 14, 2017, 4:58 PM UTC

Chriky wrote: post #19

seren wrote: post #12

Earlier quoted context omitted.

> I think if this worked as intended, no additional treatment is required. It is correct if the therapy is targeting all stem cells. I have not understood clearly from the article but it seems the treatement is targeting "living cells". Once all your 'fixed' cells have been renewed you would need another round of treatement.

I have Haemophilia A, I was actually offered a place on an earlier trial although I didn't do it. It was explained to me that you would not need further gene treatments even though it targeted adult cells. I think most cells are made by mitosis and don't come from stem cells. Here's a screenshot of the explanation I received when I was invited on the trial - https://i.imgur.com/H329Vgv.jpg > "By using BMN 270 to prov…

Post by toomuchtodo » Thu, Dec 14, 2017, 5:04 PM UTC

carbocation wrote: post #18

will_brown wrote: post #13

Earlier quoted context omitted.

>Many diseases are as simple as your eyes being brown vs blue - you simply have or lack particular proteins. I think the other half of this is the microbiome. I have been speaking to physicians about leaky gut recently and a great analogy is the protein genes are like a piano and the microbiome is like the musician. As you suggest the genes/piano is sort of the hand you are dealt (you have the gene for certain diseas…

> the microbiome is what will typically unlock those diseases or not given the gene set If true, that would be a Nobel-worthy finding. I don't think this is true, but do you have a citation supporting this for the "typical" genetic disease?

Post by openasocket » Thu, Dec 14, 2017, 5:06 PM UTC

sswaner wrote: post #8

There is a pretty strong argument that Haemophilia is the disease that has had the biggest impact on the last 100 years. Russian heir to the throne Alexis Romanov inherited the disease and his suffering and lack of treatment options destabilized his family and contributed to leadership issues culminating in Russian defeat in WW1 and the Russian revolution and the rise of the Soviet Union. Agree? If not, what disease…

Post by Chriky » Thu, Dec 14, 2017, 5:10 PM UTC

jessriedel wrote: post #23

Chriky wrote: post #19

Earlier quoted context omitted.

I have Haemophilia A, I was actually offered a place on an earlier trial although I didn't do it. It was explained to me that you would not need further gene treatments even though it targeted adult cells. I think most cells are made by mitosis and don't come from stem cells. Here's a screenshot of the explanation I received when I was invited on the trial - https://i.imgur.com/H329Vgv.jpg > "By using BMN 270 to prov…

Thanks for sharing! Can I ask what considerations went into your decision? Will you be pursuing this now that there is more evidence that it's effective?