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