What is Double DF508?

[charl72]

<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>sakasuka</b></i>

<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>charl72</b></i>



<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>jbrandonAW</b></i>



(it is one of the worst genes to get)</end quote></div>



I might as well be honest, that comment p*ssed me off. My daughter has delta F508. It is the most common mutation so when the day comes when they can find a cure for CF, need I say any more?<img src="i/expressions/face-icon-small-happy.gif" border="0"></end quote></div>



If you get pissed off that easily, you're not going to like this - you're wrong!<img src="i/expressions/devil.gif" border="0">



Allie can speak to this better than I can, but researchers are closer to correcting just one Class of CF genes...



since each class has a different defect, some are easier to correc than others. and delta F 508 isn't in the class that's closest to being corrected.</end quote></div>



I don't get p*ssed of easily. Like everyone I get p*ssed off though. I am not arguing with what you're saying, but please don't assume I get p*ssed off easily cos I don't. <img src="i/expressions/face-icon-small-tongue.gif" border="0">

 

[charl72]

Ps I am a parent of a CFer and haven't got CF myself, therefore my views and feelings are probably a lot different to yours.<img src="i/expressions/face-icon-small-confused.gif" border="0"> My daughter's Consultant told is that having her mutation was an advantage because they have done a lot of research on it like Janet said. I am sure Janet will feel the same way as me, I don't need to say anymore. Surely you can't blame us for trying to be positive and optimistic, if you were a parent you would be exactly the same (I hope anyways).<img src="i/expressions/face-icon-small-happy.gif" border="0">

Have a nice day.<img src="i/expressions/face-icon-small-cool.gif" border="0">

 

[charl72]

Ps I am a parent of a CFer and haven't got CF myself, therefore my views and feelings are probably a lot different to yours.<img src="i/expressions/face-icon-small-confused.gif" border="0"> My daughter's Consultant told is that having her mutation was an advantage because they have done a lot of research on it like Janet said. I am sure Janet will feel the same way as me, I don't need to say anymore. Surely you can't blame us for trying to be positive and optimistic, if you were a parent you would be exactly the same (I hope anyways).<img src="i/expressions/face-icon-small-happy.gif" border="0">

Have a nice day.<img src="i/expressions/face-icon-small-cool.gif" border="0">

 

[charl72]

Ps I am a parent of a CFer and haven't got CF myself, therefore my views and feelings are probably a lot different to yours.<img src="i/expressions/face-icon-small-confused.gif" border="0"> My daughter's Consultant told is that having her mutation was an advantage because they have done a lot of research on it like Janet said. I am sure Janet will feel the same way as me, I don't need to say anymore. Surely you can't blame us for trying to be positive and optimistic, if you were a parent you would be exactly the same (I hope anyways).<img src="i/expressions/face-icon-small-happy.gif" border="0">

Have a nice day.<img src="i/expressions/face-icon-small-cool.gif" border="0">

 

[SpinyChaff]

LauraLou..

That is NOT incorrect. No-one is talking about gene therapy - thats 10-15 years away or more.

sakasuka is talking about PTC124. That is likely to be the first successful treatment to correct the underlying defect. And that, unfortunately, will only treat nonesense mutations, and is only likely to be of benefit to 10% of Cfers.

 

[SpinyChaff]

LauraLou..

That is NOT incorrect. No-one is talking about gene therapy - thats 10-15 years away or more.

sakasuka is talking about PTC124. That is likely to be the first successful treatment to correct the underlying defect. And that, unfortunately, will only treat nonesense mutations, and is only likely to be of benefit to 10% of Cfers.

 

[SpinyChaff]

LauraLou..

That is NOT incorrect. No-one is talking about gene therapy - thats 10-15 years away or more.

sakasuka is talking about PTC124. That is likely to be the first successful treatment to correct the underlying defect. And that, unfortunately, will only treat nonesense mutations, and is only likely to be of benefit to 10% of Cfers.

 

[LauraLou]

<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>SpinyChaff</b></i>

LauraLou..



That is NOT incorrect. No-one is talking about gene therapy - thats 10-15 years away or more.



sakasuka is talking about PTC124. That is likely to be the first successful treatment to correct the underlying defect. And that, unfortunately, will only treat nonesense mutations, and is only likely to be of benefit to 10% of Cfers.</end quote></div>
<br/><br/>Gene therapy trials in th UK begin next year. If PTC124 is only going to treat 10% of pwcf, I doubt very much that our government would fund it.

 

[LauraLou]

<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>SpinyChaff</b></i>

LauraLou..



That is NOT incorrect. No-one is talking about gene therapy - thats 10-15 years away or more.



sakasuka is talking about PTC124. That is likely to be the first successful treatment to correct the underlying defect. And that, unfortunately, will only treat nonesense mutations, and is only likely to be of benefit to 10% of Cfers.</end quote></div>
<br/><br/>Gene therapy trials in th UK begin next year. If PTC124 is only going to treat 10% of pwcf, I doubt very much that our government would fund it.

 

[LauraLou]

<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>SpinyChaff</b></i>

LauraLou..



That is NOT incorrect. No-one is talking about gene therapy - thats 10-15 years away or more.



sakasuka is talking about PTC124. That is likely to be the first successful treatment to correct the underlying defect. And that, unfortunately, will only treat nonesense mutations, and is only likely to be of benefit to 10% of Cfers.</end quote></div>
<br/><br/>Gene therapy trials in th UK begin next year. If PTC124 is only going to treat 10% of pwcf, I doubt very much that our government would fund it.

 

[dyza]

Laura the goverment are not funding the gene therapy programme, that is being funded by the Cf trust in the UK. But there again, now that G. Brown has an interest..............?

 

[dyza]

Laura the goverment are not funding the gene therapy programme, that is being funded by the Cf trust in the UK. But there again, now that G. Brown has an interest..............?

 

[dyza]

Laura the goverment are not funding the gene therapy programme, that is being funded by the Cf trust in the UK. But there again, now that G. Brown has an interest..............?

 

[Marjolein]

Maybe the severity and the fact that delta F506 (i know it doesn't neccesarily mean the CF will be very severe) is one of the most common mutations goes hand in hand..
I always think it could well be that there are mutations that are even much more common and don't have much symptoms, but nobody knows about them because most people are relatively healthy and maybe 'just have asthma' or something and are never diagnosed with CF.

Marjolein
w/ double delta F508

 

[Marjolein]

Maybe the severity and the fact that delta F506 (i know it doesn't neccesarily mean the CF will be very severe) is one of the most common mutations goes hand in hand..
I always think it could well be that there are mutations that are even much more common and don't have much symptoms, but nobody knows about them because most people are relatively healthy and maybe 'just have asthma' or something and are never diagnosed with CF.

Marjolein
w/ double delta F508

 

[Marjolein]

Maybe the severity and the fact that delta F506 (i know it doesn't neccesarily mean the CF will be very severe) is one of the most common mutations goes hand in hand..
I always think it could well be that there are mutations that are even much more common and don't have much symptoms, but nobody knows about them because most people are relatively healthy and maybe 'just have asthma' or something and are never diagnosed with CF.

Marjolein
w/ double delta F508

 

[LauraLou]

<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>dyza</b></i>

Laura the goverment are not funding the gene therapy programme, that is being funded by the Cf trust in the UK. But there again, now that G. Brown has an interest..............?</end quote></div>

i didn't say they were

 

[LauraLou]

<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>dyza</b></i>

Laura the goverment are not funding the gene therapy programme, that is being funded by the Cf trust in the UK. But there again, now that G. Brown has an interest..............?</end quote></div>

i didn't say they were

 

[LauraLou]

<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>dyza</b></i>

Laura the goverment are not funding the gene therapy programme, that is being funded by the Cf trust in the UK. But there again, now that G. Brown has an interest..............?</end quote></div>

i didn't say they were

 

[Emily65Roses]

Okay, let's make this short and simple.

Delta F508 is NOT the worst one to get. It's a Class II. The worst genes to get, generally speaking, are Class I. Because a double DF508 is two Class II, you could be worse if you had:
ANY Class II and ANY Class I
or
Two Class I.

Gene therapy is still a ways away. As in.. the whole gene replacement. Yes, they're working on it. Is it right around the corner for purchase at your local pharmacy? No effing way.

PTC124 fixes NONSENSE mutations specifically. The only CF mutations that are nonsense are Class I. I don't care if this doesn't make sense to anyone, it's true. If you don't understand the microbiology that is involved, look it up. Don't spread misinformation by saying this PTC124 will cure any CF at all, period. It is untrue.

If you wish to read about this PTC124 in more specific detail, I suggest this link:
<a target=_blank class=ftalternatingbarlinklarge href="http://www.ptcbio.com/big/discovery1flash.html
">http://www.ptcbio.com/big/discovery1flash.html
</a>It explains why it ONLY targets the small population of CFers, and not any gene at all. It may only effect 10-15% of CFers, but generally speaking, it's the WORST Class, so yes, they're going to try and go for that one first. Class II may be more common here in the US, but it's not necessarily the most common everywhere (one of Israel's most common is w1282x, a Class I), and it's not *as a whole* deadlier than Class I. Common sense says go for the worst mutations first.

In addition, this drug is being developed BY Israel. Because Israel's most common is the w1282x (a Class I), it makes the most sense for them to target that population of CFers. It'd be like if the US started developing a drug... we'd target the Delta F508, right? Same idea. Our Delta F508 is Israel's w1282x.

Some of this stuff can be a little difficult to understand, but try to read the literature before you spout off a bunch of untrue "information" that is going to confuse not only everyone else, but also yourself.