Since announcement of the gene therapy trials, GOS has received emails from all
over the world regarding future prospects for the technique. You will
understand that it is very difficult for us to provide a detailed response to
each enquiry. We regret not being staffed to provide more assistance.
The trials carried out here are quite specific. GOS is carrying out trials of
gene therapy on two immune diseases X-SCID [x-linked Severe Combined
Immunodeficiency} and X-CGD [x-linked Chronic Granulomatous Disorder]. In the
future we hope to get approval to carry out trials on a small number of other
immune conditions - candidates for such trials include other forms of SCID, ADA
deficiency, other autosomal variants of CGD, and Wiskott-Aldrich Syndrome.
The bone marrow generates the cells of the immune system and blood. We remove
bone marrow, insert a working version of the faulty gene, and then infuse the
new cells back into the child. Early results show that the new cells grow and
provide a working immune system.
There are over 4000 genetic conditions, and at least 80 different immune
diseases alone. The technique will have to be worked out for each individual
genetic condition. We need to know the gene, be able to synthesise it and
exhaustively test the process in the lab before doing it with real children.
Furthermore, there are technical reasons why X-SCID might be an easier disease
to work on than many others.
This technique may also in the long term prove useful for diseases of the blood
such as sickle cell anaemia, and thalassemia.
So while gene therapy is extremely exciting we expect that the ranges of
diseases that are treated in this way will be limited to those described above
for the next few years. As advances are made, we are equally hopeful that we can
extend this form of therapy to other diseases in the future.
Dr Adrian Thrasher
Consultant Paediatric Immunologist
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