RNAi
Posted by living_everyday
|
RNAi February 01, 2007 12:35PM |
Registered: 17 years ago Posts: 34 |
I am a 46 year old male, at risk. No symptoms yet, thank God.
Has anybody seen any new info on Beverly Davidson? The latest articles I can find are dated 2005. In one, she mentions that human trial may begin within 5 years. I'm just wondering if that is stil true.
Hang in there. With all the people that are working on this, something has to happen soon.
There's more news which I'll probably put on the Lighthouse soon.
Progress has been made in delivery - getting RNAi to more cells (from 40 percent to 70 percent - and also on the specificity front.
The difference between Davidson's mouse study and treatment for people is that in the HD mouse, the HD gene, since it was artificially created, is very different from the mouse's normal huntingtin's protein gene. So her technique blocked the HD protein from being made while allowing the normal protein to continue to be produced in the mouse.
The same technique can't be used in people because we almost certainly need the normal protein. So what researchers have been doing is looking for single point mutations on the HD gene in people that aren't on the normal genes. They have found several and it differs from one individual to another. They are developing synthetic short interfering RNA strands that will disrupt ONLY the HD gene and not the normal gene. But people will need to go in and get their genes analyzed to see what what will work for them as individuals.
That takes care of about 70 percent of people with the HD gene. For the rest, no SNPS have been found (we're talking now about DNA samples donated for research and extrapolating from those results to the population). For the remaining group, as well as for the small group of people with two HD genes, they will have to knock down BOTH genes and then resinsert a new normal huntingtin's protein gene that differs enough that it will work and not be effected by the RNAi. So there will be a combination of RNAi AND gene therapy.
Progress has been made in delivery - getting RNAi to more cells (from 40 percent to 70 percent - and also on the specificity front.
The difference between Davidson's mouse study and treatment for people is that in the HD mouse, the HD gene, since it was artificially created, is very different from the mouse's normal huntingtin's protein gene. So her technique blocked the HD protein from being made while allowing the normal protein to continue to be produced in the mouse.
The same technique can't be used in people because we almost certainly need the normal protein. So what researchers have been doing is looking for single point mutations on the HD gene in people that aren't on the normal genes. They have found several and it differs from one individual to another. They are developing synthetic short interfering RNA strands that will disrupt ONLY the HD gene and not the normal gene. But people will need to go in and get their genes analyzed to see what what will work for them as individuals.
That takes care of about 70 percent of people with the HD gene. For the rest, no SNPS have been found (we're talking now about DNA samples donated for research and extrapolating from those results to the population). For the remaining group, as well as for the small group of people with two HD genes, they will have to knock down BOTH genes and then resinsert a new normal huntingtin's protein gene that differs enough that it will work and not be effected by the RNAi. So there will be a combination of RNAi AND gene therapy.
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