Kickas.org Forums Ankylosing Spondylitis General Discussion #1 AS Web Support Group Gene Therapy for Spondylarthropathies

In a different thread Ram suggested that in the future gene therapy may be useful in the treatment of spondylarthropathies.

Even though the mapping of the human genome continues, effecting some mutation in our DNA ("a little snip here, a little snip there") so as to get us back on track, is still quite hypothetical. On the other hand, a better understanding of protein folding is on the horizon and, in a sense, some of the experimental biologic therapies tinker with RNA, which is even closer to the messages that are encoded into our DNA.

I believe that such research will have a reverse effect than what Ram postulates. Rather than a lumping together of all the spondylarthropathies, or all "auto-immune" diseases, into one pot, I anticipate greater diversification in the future treatment of just the seronegative spondylarthropathies.


Best regards,

jcwinnie



P.S. Holly, I cleaned my cookies. Can I have my spinach now?

JC,
Though I mentioned about putting all the "auto immunes" into one pot, I was referring to the possibility that the basic cause why the immune system fails to identify the cell "self" marker may be known in future. If it turns out to be a purely genetic disease, which is not known at this time. Just like we use different antibiotics for different pathogens or families, my speculation was different gene therapy may exist for different categories of rheumatic diseases. This can also be interpreted to mean a greater diversification of diseases with a unified treatment protocol(ie gene therapy). It is only a fictitious thought!

The link on protein folding does not seem to work....


Ram

Hi Ram,

I think ? this is the link on folding proteins that JC was going for.

Ram,

Speaking of putting things in one pot, there is a new emphasis in the study of disease such as RA, AS, etc. with a combination of immunology and genetics, i.e., immunogenetics. I believe what this reflects is the interplay between our genetic complement and our immune systems. Which is another way of saying that I disagree a little with what you stated. I doubt whether in the future it will be learned that AS is "purely" a genetic disease.

Best regards,

jcwinnie

P.S. Thanks, Mig, it would seem I will never get the knack of linking to another post.

P.P.S. You know, Gerard is someone who has looked at the "AS as a genetic disease" idea.

In the lab setting, single stranded RNA is notoriously fragile because is is susceptible to degradation by enzymes known as ribonucleases (RNases) that are just everywhere, so I'm having a little bit of a faith issue that single stranded RNA could be stable enough to get used in therapy

BUT there you are, looks like folks are working on it
Here are some comments/diagrams on RNA-based approaches to gene therapy:
http://gslc.genetics.utah.edu/units/genetherapy/gtapproaches/


ANd as for protein folding, there is a current theory to explain the association of HLA B27 with AS/spondyloarthropathies that is based on the tendency of HLAB27 to MISFOLD:
http://www.jimmunol.org/cgi/content/full/163/12/6665

There is the 1999 article, to which Evelyn referred, and there is reference to protein misfolding in a 2002 review article by M.A. Khan about the Genetic Aspects of Ankylosing Spondylitis and a 2004 review article about the Immunobiology of HLA-B27. (I can attested that the latter two have high "eyes glazed information overload" potential.)

Quote:

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At school it all sounded so simple -- transcription turns DNA into RNA, and translation of RNA gives you protein. But the often forgotten third step in this process, the folding of the translated linear strand of amino acids into a fully functional three-dimensional protein, is one of the most complex challenges facing the cellular protein factory. Protein Misfolding, Nature Insights, Vol. 426, No. 6968 December, 2003.

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Best regards,

jcwinnie

CNN ran an interesting article today on medical advances on the telly. The web site didn't offer the scope of the article but offered info on the research being done on specific diseases and rectifying damaged cells through individual dna identification.
Francis Collins from the National Institue of Health and Craig Venter of Celera Geomics Inc. worked independantly on the geno code from whence Gleevec has been proven effective in leukemia. A new drug for lung cancer is in the final stages of testing. Stated in article by 2015 therapeutics of medicines will be the norm and by 2020 understanding of all diseases will have been touched. Medicines used today will be in the dust bin.
It was remarkable to witness two mice with severed spinal cords able to walk again after introducing the cell repairing gene therapy.
We are on the horizon of some of the biggest leaps in medicine.
Klem