Gene therapy

Mockingbird

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

Well they found the gene, they found out how to put the gene in- sounds pretty good to me!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!</end quote></div>

Someone correct me if I'm wrong, but I think they've known how to do that for quite a while.

I did a quick search and found an article: <a target=_blank class=ftalternatingbarlinklarge href="http://www.genome.gov/10001213">http://www.genome.gov/10001213</a>
"Gene therapy offers great promise for life-saving treatment for CF patients since it targets the cause of CF rather than just treating symptoms. Gene therapy for CF had its start in 1990, when scientists successfully corrected faulty CFTR genes by adding normal copies of the gene to laboratory cell cultures.

In 1993, the first experimental gene therapy treatment was given to a patient with CF. Researchers modified a common cold virus to act as a delivery vehicle - or "vector"- carrying the normal genes to the CFTR cells in the airways of the lung.

Subsequent studies have tested other methods of gene delivery, such as fat capsules, synthetic vectors, nose drops or drizzling cells down a flexible tube to CFTR cells lining the airways of lungs. Researchers are now testing aerosol delivery using nebulizers.

But finding the best delivery system for transporting normal CFTR genes is only one problem that scientists must solve to develop an effective treatment for CF. Scientists must also determine the life span of affected lung cells, identify the "parent cells" that produce CFTR cells, find out how long treatment should last and how often it needs to be repeated.

The first cystic fibrosis gene therapy experiments have involved lung cells because these cells are readily accessible and because lung damage is the most common, life-threatening problem in CF patients. But scientists hope that the technologies being developed for lung cells will be adapted to treat other organs affected by CF."
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I imagine this is what your doctor was talking about, but it is impossible to tell.

At any rate, I believe in the proverb "Do not boast about tomorrow, for you do not know what a day may bring forth." Or in other words, there can (and probably will) be many unforeseen obstacles and complications in the future, things which not even a doctor who is head of a research department can predict or control.

It is fine to hope for a cure, but it is wise to be prepared for a future without one.

"It is better to take refuge in the Lord
Than to trust in man.
It is better to take refuge in the Lord
Than to trust in princes [or doctors, if you prefer]" Psalm 118:8
 

Mockingbird

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

Well they found the gene, they found out how to put the gene in- sounds pretty good to me!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!</end quote></div>

Someone correct me if I'm wrong, but I think they've known how to do that for quite a while.

I did a quick search and found an article: <a target=_blank class=ftalternatingbarlinklarge href="http://www.genome.gov/10001213">http://www.genome.gov/10001213</a>
"Gene therapy offers great promise for life-saving treatment for CF patients since it targets the cause of CF rather than just treating symptoms. Gene therapy for CF had its start in 1990, when scientists successfully corrected faulty CFTR genes by adding normal copies of the gene to laboratory cell cultures.

In 1993, the first experimental gene therapy treatment was given to a patient with CF. Researchers modified a common cold virus to act as a delivery vehicle - or "vector"- carrying the normal genes to the CFTR cells in the airways of the lung.

Subsequent studies have tested other methods of gene delivery, such as fat capsules, synthetic vectors, nose drops or drizzling cells down a flexible tube to CFTR cells lining the airways of lungs. Researchers are now testing aerosol delivery using nebulizers.

But finding the best delivery system for transporting normal CFTR genes is only one problem that scientists must solve to develop an effective treatment for CF. Scientists must also determine the life span of affected lung cells, identify the "parent cells" that produce CFTR cells, find out how long treatment should last and how often it needs to be repeated.

The first cystic fibrosis gene therapy experiments have involved lung cells because these cells are readily accessible and because lung damage is the most common, life-threatening problem in CF patients. But scientists hope that the technologies being developed for lung cells will be adapted to treat other organs affected by CF."
-----------------------------------------------------------------------------



I imagine this is what your doctor was talking about, but it is impossible to tell.

At any rate, I believe in the proverb "Do not boast about tomorrow, for you do not know what a day may bring forth." Or in other words, there can (and probably will) be many unforeseen obstacles and complications in the future, things which not even a doctor who is head of a research department can predict or control.

It is fine to hope for a cure, but it is wise to be prepared for a future without one.

"It is better to take refuge in the Lord
Than to trust in man.
It is better to take refuge in the Lord
Than to trust in princes [or doctors, if you prefer]" Psalm 118:8
 

dasjsmum

New member
I think there's a few different things being spoken about here:

The cf gene was isolated in 1989, ages ago...they thought there would be a cure etc, as Emily said...

As Amy mentioned, the drug in the pipe line only works for a small percentage of the cf population, mainly of Jewish descent (from what I understand, 'nonsense mutations' there are different ones apparently, not just Jewish descent, but that's the main one...I could be wrong though...).

The UK are working on a gene therapy which has just had 40million pounds (I guess) injected into it, and looks very promising...that is still a long way off though.

Anyhow, keep the faith, hope is a great thing to have, it energises and enthuses, we all still have faith that at least a great treatment is on its way, the sooner the better.

I guess I dont really see a cure as such, but a great treatment which addresses the major problem areas and allows for 'normal' life...that would be fantastic.
 

dasjsmum

New member
I think there's a few different things being spoken about here:

The cf gene was isolated in 1989, ages ago...they thought there would be a cure etc, as Emily said...

As Amy mentioned, the drug in the pipe line only works for a small percentage of the cf population, mainly of Jewish descent (from what I understand, 'nonsense mutations' there are different ones apparently, not just Jewish descent, but that's the main one...I could be wrong though...).

The UK are working on a gene therapy which has just had 40million pounds (I guess) injected into it, and looks very promising...that is still a long way off though.

Anyhow, keep the faith, hope is a great thing to have, it energises and enthuses, we all still have faith that at least a great treatment is on its way, the sooner the better.

I guess I dont really see a cure as such, but a great treatment which addresses the major problem areas and allows for 'normal' life...that would be fantastic.
 

dasjsmum

New member
I think there's a few different things being spoken about here:

The cf gene was isolated in 1989, ages ago...they thought there would be a cure etc, as Emily said...

As Amy mentioned, the drug in the pipe line only works for a small percentage of the cf population, mainly of Jewish descent (from what I understand, 'nonsense mutations' there are different ones apparently, not just Jewish descent, but that's the main one...I could be wrong though...).

The UK are working on a gene therapy which has just had 40million pounds (I guess) injected into it, and looks very promising...that is still a long way off though.

Anyhow, keep the faith, hope is a great thing to have, it energises and enthuses, we all still have faith that at least a great treatment is on its way, the sooner the better.

I guess I dont really see a cure as such, but a great treatment which addresses the major problem areas and allows for 'normal' life...that would be fantastic.
 

dasjsmum

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

I for one can understand the excitement of the possibility, but also I can understand the wait and see aspect as well. I tell everyone that I run across that when my little girl is 10 or maybe 15 that CF will be a like diabetes. I don't think I'm wrong for saying that. Now, I'm not a Doctor by any means, but I do work with Doctors and labs. each day that work with CF. And sure I don't think that it is out of the realm of thinking to think that CF will take a huge turn in advancement in the next few years. My daughter is 3, she was dx at 1.5, and during that time look what has been pushed through and what has been developed. Everyones hope hinges on the gene therepy, because that seems the "most likely" cure. Those of you that have been hearing the cure is coming the cure is coming sure may feel like it is a broken record, and sure you may get tired of newcomers or people who post here not as often saying I heard this or I heard that. Who is to say that they may not be right! For those internet sites to be posted doesn't a doctor like the poster is referring to have to pass that information along. They don't let some Joe Blow writer just make it up. Let's remember we all are here for the same purpose and that is to make sure all who are affected by CF (CFer, parent, wife, husband, son, daughter, best friend) can talk to each other and not lose hope. I don't know what it is like to have CF, but I do remember to this day the feeling I had when I was holding my little girl when the Doctor told me she had CF. It was something I "thought" at the time I couldn't take away from her or make it better, but the more I got involved with the Doctors and labs I work with, the more I realized that it is not unlikely that a cure won't be found by the time she 15-20 yrs. if not a cure, then a longterm treatment that will help her live a normal life to 65,75,85 or even 95 years of age. CF is not what it was 5, 10, 15, 20 years ago, sure it is the same disease. But as time change and medicine advances so does the hope of CF. Look at the pipeline I ask you, and compare it to 1989 or even 1999, and tell me that we should not hope. Sorry for the spelling!



Chuck father to Avery 3 years of age with Cystic Fibrosis DF508/DF508, and Rhett 2 years of age carrier--Knoxville, TN</end quote></div>

Hi there

I dont want to be negatively painful, but could you please break up your posts with paragraph spaces?

Unfortunately, when I see a post like this, I usually dont read it because it is too difficult...if you use spaces more, it is much easier to read your post.

Thanks very much
 

dasjsmum

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

I for one can understand the excitement of the possibility, but also I can understand the wait and see aspect as well. I tell everyone that I run across that when my little girl is 10 or maybe 15 that CF will be a like diabetes. I don't think I'm wrong for saying that. Now, I'm not a Doctor by any means, but I do work with Doctors and labs. each day that work with CF. And sure I don't think that it is out of the realm of thinking to think that CF will take a huge turn in advancement in the next few years. My daughter is 3, she was dx at 1.5, and during that time look what has been pushed through and what has been developed. Everyones hope hinges on the gene therepy, because that seems the "most likely" cure. Those of you that have been hearing the cure is coming the cure is coming sure may feel like it is a broken record, and sure you may get tired of newcomers or people who post here not as often saying I heard this or I heard that. Who is to say that they may not be right! For those internet sites to be posted doesn't a doctor like the poster is referring to have to pass that information along. They don't let some Joe Blow writer just make it up. Let's remember we all are here for the same purpose and that is to make sure all who are affected by CF (CFer, parent, wife, husband, son, daughter, best friend) can talk to each other and not lose hope. I don't know what it is like to have CF, but I do remember to this day the feeling I had when I was holding my little girl when the Doctor told me she had CF. It was something I "thought" at the time I couldn't take away from her or make it better, but the more I got involved with the Doctors and labs I work with, the more I realized that it is not unlikely that a cure won't be found by the time she 15-20 yrs. if not a cure, then a longterm treatment that will help her live a normal life to 65,75,85 or even 95 years of age. CF is not what it was 5, 10, 15, 20 years ago, sure it is the same disease. But as time change and medicine advances so does the hope of CF. Look at the pipeline I ask you, and compare it to 1989 or even 1999, and tell me that we should not hope. Sorry for the spelling!



Chuck father to Avery 3 years of age with Cystic Fibrosis DF508/DF508, and Rhett 2 years of age carrier--Knoxville, TN</end quote></div>

Hi there

I dont want to be negatively painful, but could you please break up your posts with paragraph spaces?

Unfortunately, when I see a post like this, I usually dont read it because it is too difficult...if you use spaces more, it is much easier to read your post.

Thanks very much
 

dasjsmum

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

I for one can understand the excitement of the possibility, but also I can understand the wait and see aspect as well. I tell everyone that I run across that when my little girl is 10 or maybe 15 that CF will be a like diabetes. I don't think I'm wrong for saying that. Now, I'm not a Doctor by any means, but I do work with Doctors and labs. each day that work with CF. And sure I don't think that it is out of the realm of thinking to think that CF will take a huge turn in advancement in the next few years. My daughter is 3, she was dx at 1.5, and during that time look what has been pushed through and what has been developed. Everyones hope hinges on the gene therepy, because that seems the "most likely" cure. Those of you that have been hearing the cure is coming the cure is coming sure may feel like it is a broken record, and sure you may get tired of newcomers or people who post here not as often saying I heard this or I heard that. Who is to say that they may not be right! For those internet sites to be posted doesn't a doctor like the poster is referring to have to pass that information along. They don't let some Joe Blow writer just make it up. Let's remember we all are here for the same purpose and that is to make sure all who are affected by CF (CFer, parent, wife, husband, son, daughter, best friend) can talk to each other and not lose hope. I don't know what it is like to have CF, but I do remember to this day the feeling I had when I was holding my little girl when the Doctor told me she had CF. It was something I "thought" at the time I couldn't take away from her or make it better, but the more I got involved with the Doctors and labs I work with, the more I realized that it is not unlikely that a cure won't be found by the time she 15-20 yrs. if not a cure, then a longterm treatment that will help her live a normal life to 65,75,85 or even 95 years of age. CF is not what it was 5, 10, 15, 20 years ago, sure it is the same disease. But as time change and medicine advances so does the hope of CF. Look at the pipeline I ask you, and compare it to 1989 or even 1999, and tell me that we should not hope. Sorry for the spelling!



Chuck father to Avery 3 years of age with Cystic Fibrosis DF508/DF508, and Rhett 2 years of age carrier--Knoxville, TN</end quote></div>

Hi there

I dont want to be negatively painful, but could you please break up your posts with paragraph spaces?

Unfortunately, when I see a post like this, I usually dont read it because it is too difficult...if you use spaces more, it is much easier to read your post.

Thanks very much
 

NoExcuses

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



I dont want to be negatively painful, but could you please break up your posts with paragraph spaces?


</end quote></div>


I love you for saying that.

i'm trying to spread the word about that on this board as well.
 

NoExcuses

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



I dont want to be negatively painful, but could you please break up your posts with paragraph spaces?


</end quote></div>


I love you for saying that.

i'm trying to spread the word about that on this board as well.
 

NoExcuses

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



I dont want to be negatively painful, but could you please break up your posts with paragraph spaces?


</end quote></div>


I love you for saying that.

i'm trying to spread the word about that on this board as well.
 

purplemartin

New member
I agree with the whole space thing..........but this forum will NOT let me space my paragraphs at ALL! Maybe she is having the same problem????? As far as the original post, I agree with most. As a parent, I would absolutely LOVE to see a cure sometime in my son's life, but I do not count on that cure! The common cold, breast cancer, and thousands of other common disease still have no cure. Breast cancer awareness receives so much more money (money=cure) than the CFF does toward research and they have yet to find a cure themselves. Like I said I hope one day we do see a cure, and we are on the path of doing so. However, my faith is in better treatments at this point, hopefully one day leading to that cure!
 

purplemartin

New member
I agree with the whole space thing..........but this forum will NOT let me space my paragraphs at ALL! Maybe she is having the same problem????? As far as the original post, I agree with most. As a parent, I would absolutely LOVE to see a cure sometime in my son's life, but I do not count on that cure! The common cold, breast cancer, and thousands of other common disease still have no cure. Breast cancer awareness receives so much more money (money=cure) than the CFF does toward research and they have yet to find a cure themselves. Like I said I hope one day we do see a cure, and we are on the path of doing so. However, my faith is in better treatments at this point, hopefully one day leading to that cure!
 

purplemartin

New member
I agree with the whole space thing..........but this forum will NOT let me space my paragraphs at ALL! Maybe she is having the same problem????? As far as the original post, I agree with most. As a parent, I would absolutely LOVE to see a cure sometime in my son's life, but I do not count on that cure! The common cold, breast cancer, and thousands of other common disease still have no cure. Breast cancer awareness receives so much more money (money=cure) than the CFF does toward research and they have yet to find a cure themselves. Like I said I hope one day we do see a cure, and we are on the path of doing so. However, my faith is in better treatments at this point, hopefully one day leading to that cure!
 

purplemartin

New member
It will let me space in my signature for some reason, but not in my replies. I have tried to figure this one out but gave up. I would loooove to be able to space my paragraphs though. I wonder if I put in the HTML code if it will space........Lets see?



Did it work.

It did, YAY! Just stinks I have to post it between paragraphs though.
 

purplemartin

New member
It will let me space in my signature for some reason, but not in my replies. I have tried to figure this one out but gave up. I would loooove to be able to space my paragraphs though. I wonder if I put in the HTML code if it will space........Lets see?



Did it work.

It did, YAY! Just stinks I have to post it between paragraphs though.
 

purplemartin

New member
It will let me space in my signature for some reason, but not in my replies. I have tried to figure this one out but gave up. I would loooove to be able to space my paragraphs though. I wonder if I put in the HTML code if it will space........Lets see?



Did it work.

It did, YAY! Just stinks I have to post it between paragraphs though.
 
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