Huntingdons Disease - successfully treated for the 1st time
Discussion
Truly groundbreaking, You can see why the team would be emotional. Working with families afflicted by this awful disease and seeing them decline, helpless to intervene, and now having created a meaningful therapy. I bet they are walking on clouds today! 
Someone with enough posts please point this out to the idiots on the COVID thead in NP&E, this is what comes from mRNA technology!
Someone with enough posts please point this out to the idiots on the COVID thead in NP&E, this is what comes from mRNA technology!
Amazing stuff! I have a friend who is on a trial for similar surgery for Parkinson's so really hopeful she can get the same sort of results.
(It's randomised so she has a 1/3 chance of being in the control group and only receive the 12 hour anaesthetic and a hole in her head. Brave of people to take part, albeit they are desperate. )
(It's randomised so she has a 1/3 chance of being in the control group and only receive the 12 hour anaesthetic and a hole in her head. Brave of people to take part, albeit they are desperate.
)Great discovery if it works safely but there is a viral vector involved, at least it looks like it, and there are risks with that delivery method that have stymied gene therapy trials for other diseases. Any peer reviewed papers on this?
https://www.clinicaltrials.gov/study/NCT04120493
https://www.clinicaltrials.gov/study/NCT04120493
Edited by Yahonza on Wednesday 24th September 17:56
Yahonza said:
Great discovery if it works safely but there is a viral vector involved, at least it looks like it, and there are risks with that delivery method that have stymied gene therapy trials for other diseases. Any peer reviewed papers on this?
https://www.clinicaltrials.gov/study/NCT04120493
What are the risks you allude to in other trials?https://www.clinicaltrials.gov/study/NCT04120493
Edited by Yahonza on Wednesday 24th September 17:56
Huntington’s disease has such a bleak outlook that they would need to be pretty major to outweigh the benefits than appear to come from this therapy in this early trial.
Origami said:
Yahonza said:
Great discovery if it works safely but there is a viral vector involved, at least it looks like it, and there are risks with that delivery method that have stymied gene therapy trials for other diseases. Any peer reviewed papers on this?
https://www.clinicaltrials.gov/study/NCT04120493
What are the risks you allude to in other trials?https://www.clinicaltrials.gov/study/NCT04120493
Edited by Yahonza on Wednesday 24th September 17:56
Huntington s disease has such a bleak outlook that they would need to be pretty major to outweigh the benefits than appear to come from this therapy in this early trial.
Not that this will stop developments of the new treatment for Huntington's and other CNS disorders, but may limit how widespread these become. As long as patients are advised of the potential risks and the risks are quantified.
Thanks.
Some of this is the same kind of my e response that you get with any viral infection, some is particular not to the viral vector but rather to the expressed trans gene and should therefore be able to be managed by altering the sequence with this in mind and there is promising work on adjacent immunosupression to minimise both effects.
Clearly like any therapy it needs careful evaluation but on the face of it, it is an amazing step forward for a previously untreatable disease.
I wonder how far the USA is falling behind given the current administrations opposition to funding for gene therapies and mRNA in particular
Some of this is the same kind of my e response that you get with any viral infection, some is particular not to the viral vector but rather to the expressed trans gene and should therefore be able to be managed by altering the sequence with this in mind and there is promising work on adjacent immunosupression to minimise both effects.
Clearly like any therapy it needs careful evaluation but on the face of it, it is an amazing step forward for a previously untreatable disease.
I wonder how far the USA is falling behind given the current administrations opposition to funding for gene therapies and mRNA in particular
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