Discussion Forums > Working Towards a Cure

gene therapy

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souly:
Please see the below message from Pat.
My prayer with the team and the patients.
Souly

Dear family,
 
Things are going wonderfully.
 
We have completed the stem cell mobilisation in the first 3 patients. The per centage of cells that we are entering is in the first batch from the lab is HIGH. This is great news.
 
3 months for our first patient.
 
pat

Andy Battaglia:
What this means?

A major obstacle in using stem cells has been collecting a sufficient quantity of a patient's stem cells from the blood in which to introduce the good genes. This obstacle has now been overcome and a more than sufficient quantity of stem cells can now be "mobilized" from the bone marrow into the blood where they can be collected for later use with the introduced genes. This really is a most important step in the process and I agree with Pat that we are now very close to seeing a human cured with gene therapy. It will be exciting to hear what Dr Sadelain has to say at the upcoming conference in NYC.

Manal:
Thank you for the explaination Andy. I thought that this is an ''easy'' procedure since it is done with the donor in BMT, may be there is a different way

Anyway can not wait to know what Dr. Sadelain will say :pray :pray :pray :pray

manal

Andy Battaglia:
Manal,

After hearing the various presentations at the gene therapy symposium in February, I have to say that it is anything but easy. The road to where they are now has been a long difficult road. Understanding the factors involved in making this happen, just makes this latest development even more encouraging. For gene therapy to work, a sufficient quantity of stem cells must be produced in the bone marrow so that a large enough quantity of the good genes can be implanted, so that the cells with the good beta thal genes can produce enough hemoglobin to free the patient from transfusions. These new good red blood cells must also be able to continue to reproduce in sufficient amounts to maintain the hemoglobin to a range of around 10. I believe we will soon see the fruits of these long years of research and development.

Sharmin:

--- Quote ---These new good red blood cells must also be able to continue to reproduce in sufficient amounts to maintain the hemoglobin to a range of around 10. I believe we will soon see the fruits of these long years of research and development.
--- End quote ---

Praying for this to happen   :pray :pray :pray :pray :pray :pray :pray

Sharmin

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