7 November 1997

Apparent pt location: USA

I have an incurable, untreatable neuromuscular disease, probably autosomal dominant limb-girdle muscular dystrophy.

1. How does the body recognize that a gene is dominant?
2. Is there something in the sequence of the coding for protein synthesis or is it more likely in the genetic material between genes?
3. What is the current state of research in discovering how to inactivate a defective dominant gene?

I recently read about three researchers trying to change the way a cell "reads" a defective gene using antisense oligonucleotides. (I need a definition of that term.)
Is such research still in its infancy?

Last December when Vical received a patent for their method of injecting naked DNA into muscle cells, it sounded like exactly what I need. Of course, my exact gene defect has not yet been identified, much less located, sequenced, and reproduced.

Any information would be greatly appreciated.