Gene Therapy

[sunnygirl]

Could you please give an example of how gene therapy would be used to treat a disese??

[MrMistery]

Well, gene therapy basically introduces some DNA into your own DNA. Any desease that can be cured by that could theoretically be cured by gene therapy. The first desease to be cured through this method was SCID.

[sunnygirl]

Ok so gene therapy can cure SCID? I was told that there wasnt a definant cure for it. In order for it to work you would have to keep on injecting the good DNA?

[MrMistery]

Yes that is true. But this is one kind of gene therapy. Although not a deffinitive cure, it is a start.

[sunnygirl]

Ok thanks for the info i found to be much use to me, thanks again. but one last thing, can you insert any normal gene into the cell? or is it only usefull with certian ones?

[truman]

in theory you can insert any normal gene into the cell, but there are some technological problems

[weesper]

I will just post some more on SCID here; SCID (severe combined immunodeficiency) was chosen as a proff of principle because it is possible to do ex vivo gene therapy, that is one can isolate or enrich for putative stem cells from a bone marrow tap and then transduce these cells (that is inject a healthy copy of the gene in question) into these cells (of course you're not literally injecting it, a retroviral vector was used). This retroviral vector will insert the healthy gene randomly into the patients DNA in several of the presumptive stem cells which should now start producing the protein that the gene codes for. After checking for whether the cells actually do produce the protein you can then re-infuse the cells back into the patient which find their way back into the bone marrow.

Problem is that several patients have developed leukemias as a consequence of this therapy. It turned out that the retroviral vector inserts into actively transcribed genes preferentially and that the defective gene can play a role as an oncogene. Both factors combined may for a dangerous combination and these are issues that are currently being worked on.

The fact remains that it is a life-saving and very gracious technique of really curing disease (apart from the very severe side effects as said, these patients we're actually cured). I hope this contributes to the discussion take care mxj