Gene Therapy

http://www.foxnews.com/science/index.html

Please check out this link. An american just won the Nobel prize for his studies on how cells use information from genes to make certain proteins.

There were also two other Americans who share the Nobel Prize for their studies on Gene Therapy.

Lets hope people, this is the break we've been waiting for.

Hi Christine,

I belive that you are referring to the Nobel Prize for Chemistry - American Roger D. Kornberg won the prize in chemistry for his studies of how cells take information from genes to produce proteins, a process that could provide insight into defeating cancer and advancing stem cell research.

This in conjunction with the research that resulted in the Nobel Prize for Medicine - Andrew Z. Fire and Craig C. Mello for discovering a powerful way to turn off the effect of specific genes (http://www.msnbc.msn.com/id/15101676/) - definitely raises hopes for gene therapy/stem cell reasearch being a possible cure for Thalassemia. This seems to be the first step towards this direction.

Keeping my fingers crossed .....

Bostonian

Hi Christine,

Read the link. I am praying like mad that something like that is quickly implemented in hospitals. Then Lauryn and I can and all of us can have a real party  . I am getting really hopeful now. Keeping my fingers crossed.

Take care of yourself and Lauryn.

Regards,
Namitha

Folks,

I may have some news..

I recently wrote a email to  Dr.Punam Malik, MD, Associate Professor of Pediatrics, Childrens Hospital Los Angeles, Associate Professor of Pediatrics, Cincinnati Children's Hospital Medical Center and was thrilled to see a response which made me feel better.

Dr Punam says with the correction of mouse models of thalassemia and human models of thalassemia major (by their group), gene therapy for thalassemia is on the horizon. Preclinical studies are underway by her group and others.
 
Lets stay tuned on the progress in gene therapy, since it will likely become the therapy of the future. 

I have attached a study conducted by Dr Punam and the group on Gene therapy. This could be slightly outdated but Dr Punam's email to me recently has given me a lot of faith to see a cure for Thal thru Gene therapy for my 6 yr old angel- we call her "Aanchal".

cheers

Deepak
09880196272

I remember chating with Lisa in 2000. And I am sure her dreams would come true some day! Thanks Andy and the community for your support!

Hi Deepak,

I would like to add a further note to this. I found this on the TIF website but unfortunately, no more information was given, but this is a quite dramatic development.

From  http://www.thalassaemia.org.cy/

First gene therapy treatment for Thalassaemia has been applied to a patient in France this month. A total of 10 volunteers are due and the results of this trial are eagerly awaited by the Thalassaemia community.

I would say waiting eagerly is an understatement. As you see from the results of our ongoing poll at

http://www.thalassemiapatientsandfriends.com/index.php?topic=144.0

Gene therapy is the number one response as to research priorities for thalassemia. It is the one possibility for a cure that seems both attainable and which also holds promise for adult thalssemics. We will be watching this trial with great hope.

For those members who have not voted in this poll, please do so. The results of this poll are watched by decision makers regarding the directions of research and the opinions of those who have to deal with thal hold great weight.

Hi Deepak and Andy,

God bless you! Please keep us updated on any news of a breakthrough. Andy is right. We need to vote so those docs and researchers know that we are desperate  . At least I am!

Regards,
Namitha