somatic cell gene therapy

davis at davis at
Sun Jan 22 17:24:11 EST 1995

In article <ugm2ejs.1.000F0B9B at>, ugm2ejs at (E.J. 
Saunders) writes:

> I am a 3rd year medical student intercalating in biochem/ molecular biology.
> I am currently researching the field of ex vivo somatic cell gene therapy "The 
> potential of using skin cells". I have looked at all the mainstream stuff and 
> it appears that even the "leaders" eg; Dusty Miller, I.Verma etc. have rather 
> given up on using fibroblast/keratinocyte primary cells as feasible gene 
> product delivery systems due to problems with in vivo expression (gene 
> inactivation?) and seem to be defecting to myoblasts instead!  Can anyone give
> me some optimistic news concerning skin cell research in this area.
> A group in China have actually done some stage I clinical trials using 
> modified fibroblasts/collagen gel injections in an effort to deliver factor IX 
> - the results seemed promising.  I can't believe that the "skin approach" is a 
> lost cause......Do you?

This area remains very interesting.  Have a look at:

Lotze MT and Rubin JT.  Gene therapy of cancer: a pilot study of
IL-4-gene-modified fibroblasts admixed with autologous tumor to elicit an
immune response.  Human Gene Therapy 5: 41-55, 1994.

Veelken H et al.  Primary fibroblasts from human adults as target cells for ex
vivo transfection and gene therapy.  Human Gen Therapy 5: 1203-1210, 1994.

It depends what you want to do.  A vaccination strategy such as in the first 
paper above does not require long-term gene expression, so this system is 
ideal.  If you want stable long-term expression, it is difficult to get 
this in fibroblasts as they tend to turn it off.  Some groups are looking
at using different promoters which might be better.

Ian Davis					davis at
Ludwig Institute for Cancer Research
Melbourne Tumour Biology Branch
Melbourne, Australia.

> All opinions,suggestions,references etc would be most welcome.
> It is a fascinating area and I want to gain the broadest, most up-to-date
> view on the subject!
> Cheers!
>                       Emily
> e-mail address:ugm2ejs at 

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