A lot of information is now becoming available, connecting specific genes with specific diseases. Sometimes the disease is due to specific mutations on these genes. How do you use this information?
One possible method seems to be to produce vaccines to deactivate the proteins produced by the defective gene. This is obviously easier said than done! Immunotherapy for cancer does something similar. However, it is certainly worth discussing general techniques for producing specific vaccines to deal with diseases known to be caused by specific mutations. Would someone guide us to publications relevant to this idea?
Srinivasan Ramani
You may wish to read
https://en.wikipedia.org/wiki/DNA_vaccination
It does not report any significant progress in this direction so far. If the defective gene can be made to function in some environment like the duck egg and if antibodies to the protein created can be harvested, we may get a serum that may have therapeutic value. Creating antigens in sufficient quantitity in a form safe enough to inject is another possibility.
All we can ask for at this stage is a set of references to relevant publications, to stimulate wider thinking.
It is a good thought. I also often have wild idea on "vaccine" to prevent / treat hereditary disease associated with mutations. But, the major question often come to my mind is prevalence/incidence. I think factors such as less frequency (worldwide) or regional/race specificity plays a key role in development of such vaccines. It is well known that design and development of a vaccine is time and massive money consuming process... If we consider human efforts, time and phase trails it will be too difficult to deliver a product (for one mutation) to a society. Yet, we don't have concepts on vaccine for targeted population or geographic settings. Both factors plays a major role in disease related with mutations. I think this is the major reason no or very few efforts (if ) made on vaccine based approaches to manage mutation associated diseases/disorders..
we cannot let even a single individual to die in vain... But, making huge efforts for one rare genetic mutation does not seems logical to me.. Hence, now I started to think about substituting it with allogenic HSCT procedures looks cheaper and easier to perform/deliver productive approaches to the society...
I may be wrong but your thoughts are notable. Major problems such as thalassemia can be targeted according to your thought... .
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