in order for gene therapy to succeed a selective advantage must be gained ! 1. french child with...
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In order for gene therapy to succeed a selective advantage
must be gained !
1. French child with x-linked SCID –
improved after spontaneous reverse
mutation.
2. In CGD and LAD no advantage to
transfected cells – all trails failed !
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Gene therapy in x-linked SCID
1. C is ubiqentously expressed from a very immature stage up to mature cells.
2. C is continuously transcribed in
lymphocytes in both resting and activated
states. No tight control is needed.
3. C is function provide survival and
proliferative signal strong growth advantage!
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NATURE VOL 433 10 FEBRUARY 2005
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Gamma chain gene therapy cause lymphoma!!!! Nature 2006
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Reply from: London, Hanover, Cincinnati, NIH, Tokyo, Memphis, Glasgow Paris
1. Overexpression to artificially high level
2. Only one lymphoma after 1.5 in mice
3. IL2RG by itseld is not oncogenic
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WHEN INTERFIRING WITH NATURE THERE IS NO BENEFITS OR FAULTS – THERE ARE CONSEQUENCES.
New England Journal of Medicine 2004