This is amazing, incredibly expensive and brand new but absolutely amazing.
It starts with a safe virus that has been altered to contain a specially designed sequence of DNA.
This is infused deep into the brain using real-time MRI scanning to guide a microcatheter to two brain regions - the caudate nucleus and the putamen. This takes 12 to 18 hours of neurosurgery.
The virus then acts like a microscopic postman – delivering the new piece of DNA inside brain cells, where it becomes active.
This turns the neurons into a factory for making the therapy to avert their own death.
This is amazing, incredibly expensive and brand new but absolutely amazing.
I'm surprised the effects lasted as long as they did. I don't know much about gene therapy, but longevity is not something I usually see associated with it. Edit: they mention because it's brain...
I'm surprised the effects lasted as long as they did. I don't know much about gene therapy, but longevity is not something I usually see associated with it.
Edit: they mention because it's brain cells, which last much longer, that it'll 'last for life', very cool.
This is fantastic news for folks who have the gene. The 75% slowdown is magnificent: it's decades more of good quality life, which means who knows if they'll live well enough to see a complete cure.
This is fantastic news for folks who have the gene. The 75% slowdown is magnificent: it's decades more of good quality life, which means who knows if they'll live well enough to see a complete cure.
I am hopeful that when they look at stage zero patients - before any negative effects have happened - they'll find this prevents it from developing entirely. If you never get that toxic protein...
I am hopeful that when they look at stage zero patients - before any negative effects have happened - they'll find this prevents it from developing entirely. If you never get that toxic protein maybe it'd be a pre-emptive cure even if not a post-symptomatic one.
Yeah I hope the first prevention trial is a success! With my limited understanding of how this works, the people already have this gene and the cells pre-emptively making this microRNA should work...
Yeah I hope the first prevention trial is a success! With my limited understanding of how this works, the people already have this gene and the cells pre-emptively making this microRNA should work right?
I'm also very happy to hear of the precedence, of national health care paying for haemophilia B. gene therapy, so good chance this one will be covered too. It's good for the nation: a few million £ is cheap compared to what a healthy citizen can contribute to during their lifetime, the amount saved from medical care in the system, saved from various specialists, as well as the loss productivity from family having to care for the person.
Great question, I think so. But I'm not a .... Custom-built-mRNA-delivering-virologist Whatever the technical term is. Looks like neurodegenerative specialty for neurologist/neuroscientist
Great question, I think so. But I'm not a .... Custom-built-mRNA-delivering-virologist
Whatever the technical term is.
Looks like neurodegenerative specialty for neurologist/neuroscientist
Would be great if they could work on the transmission of or correction of the mutation itself as well, as it is an autosomal dominant disease (any copy of the mutation from either parent will...
Would be great if they could work on the transmission of or correction of the mutation itself as well, as it is an autosomal dominant disease (any copy of the mutation from either parent will manifest the disease). That said, amazing progress!
I mean that is definitely a different type of gene therapy or embryonic selection with its own complications and considerations. Definitely different teams than the treatment folks. If there's a...
I mean that is definitely a different type of gene therapy or embryonic selection with its own complications and considerations. Definitely different teams than the treatment folks.
If there's a treatment though folks might be less inclined not to get tested because they don't want the anxiety of knowing it's coming.
This is amazing, incredibly expensive and brand new but absolutely amazing.
This is fantastic news. My mother was a social worker and always said Huntington's was the worst disease that scared her the most. Science is amazing.
I'm surprised the effects lasted as long as they did. I don't know much about gene therapy, but longevity is not something I usually see associated with it.
Edit: they mention because it's brain cells, which last much longer, that it'll 'last for life', very cool.
This is fantastic news for folks who have the gene. The 75% slowdown is magnificent: it's decades more of good quality life, which means who knows if they'll live well enough to see a complete cure.
I am hopeful that when they look at stage zero patients - before any negative effects have happened - they'll find this prevents it from developing entirely. If you never get that toxic protein maybe it'd be a pre-emptive cure even if not a post-symptomatic one.
Yeah I hope the first prevention trial is a success! With my limited understanding of how this works, the people already have this gene and the cells pre-emptively making this microRNA should work right?
I'm also very happy to hear of the precedence, of national health care paying for haemophilia B. gene therapy, so good chance this one will be covered too. It's good for the nation: a few million £ is cheap compared to what a healthy citizen can contribute to during their lifetime, the amount saved from medical care in the system, saved from various specialists, as well as the loss productivity from family having to care for the person.
Great question, I think so. But I'm not a .... Custom-built-mRNA-delivering-virologist
Whatever the technical term is.
Looks like neurodegenerative specialty for neurologist/neuroscientist
Would be great if they could work on the transmission of or correction of the mutation itself as well, as it is an autosomal dominant disease (any copy of the mutation from either parent will manifest the disease). That said, amazing progress!
I mean that is definitely a different type of gene therapy or embryonic selection with its own complications and considerations. Definitely different teams than the treatment folks.
If there's a treatment though folks might be less inclined not to get tested because they don't want the anxiety of knowing it's coming.