in order for gene therapy to succeed a selective advantage must be gained ! 1. french child with...
TRANSCRIPT
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 !
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!
NATURE VOL 433 10 FEBRUARY 2005
Gamma chain gene therapy cause lymphoma!!!! Nature 2006
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
WHEN INTERFIRING WITH NATURE THERE IS NO BENEFITS OR FAULTS – THERE ARE CONSEQUENCES.
New England Journal of Medicine 2004