Gene Therapy On the Up?

[waterboy1]

Gene Therapy On the Up?

Is gene therapy on the positive note now that there have not been deaths and the research has continued on a positive note?

also the successes of many gene therapy clinical trials such as X-SCID humans (bubble boys) working makes me think things may be going well for human gene therapy.

On the other hand there has been no progress on the front of corrective gene therapy (much better obviously) in which the defective gene is changed with the corrected gene, instead of simply integrating corrected copies anywhere in the genome.

just my 2 cents....

[koukou2]

I would say it is as many disease have been "cured"....

there have been problems with the bubble boy gene therapies as some have developed cancer from the gene therapy....

viruses inserting into the genome is not a good thing...

[MicroKiller]

i would think that by altering the genes with therapy.. the possibility of mutation to occur may lead to unwanted side effects... i am not too confident of gene therapy as yet.. anyway thts wht i think...

[darkwng]

Yes, I think we have to wait more and see results!

[aftabac]

Gene therapy is good in some diseases, just like SCID because in this case the cell that have a mutated gene circulate in the body so we can pick cells and manupulate them outside, grow them and put them back into the body, so the normal gene give product that can lead to normal phenotypes, but gene therapy for complex disorders like cancer in whcih many genes are mutated is a tough task so far,
another alternative to gene therapy is use of RNAi to block the gene that give the mutated product, but this is successful in those cases in which one copy of that gene is normal (un mutated).
Although we are hopping for better future of gene therapy

aftab

[Frank]

I would agree that gene therapy is on the up. I know of promising results with the mouse model for Duchenne muscular dystrophy. A number of clinical trials are ongoing. I think the 3 most common muscular dystrophies (Duchenne, Myotonic Dystrophy and Facioscapulohumeral Dystrophy) will have treatments within 10 years. They won't necessarily be full cures but still major improvements.
Frank from The Netherlands

[Cebe]

Not sure gene therapy is on the up. We should never forget that being on the up for a therapeutical strategy means curing (or improving the health) of patients and the gene therapy does not fulfill this criteria. Please note I am convinced that gene therapy may be a good approach to help patients but it is (still) now a too young and naive field.

[Frank]

Originally Posted by Cebe

We should never forget that being on the up for a therapeutical strategy means curing (or improving the health) of patients and the gene therapy does not fulfill this criteria.

I disagree, we have SCID and malignant melanoma that can now be cured/improved with gene therapy. And besides, I think that showing potential is enough to classify a therapy as being on the up. And gene therapy is showing potential, as judged by the succes with SCID and malignant melanoma and numerous trials in animals and ongoing clinical trials.

[Frank]

A list of companies involved in gene therapy. I have no idea if this is an exhaustive list. At the time of writing there were approximately 120 companies listed.

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[Frank]

As an 8-year-old boy becomes the first U.S. gene therapy patient for Duchenne muscular dystrophy, one of the researchers behind the therapy talks about the 20 years it took to get there, and what lies in the future.

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