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(HemAware) gene therapy trials Gene Therapy FAQs Q: What is gene
therapy? A. Gene therapy is an exciting experimental technique
that seeks to provide the body with the genetic information
(DNA) it lacks so that it can perform normal functions. Most
gene therapy strategies use a vector, such as modified virus, to
attempt to deliver a good copy of a gene to a cell that contains
a defective gene. Scientists hope that the transferred gene will
then restore the normal functions. For people with hemophilia,
this means that clotting factor genes inserted into the body
would enable normal clotting factor proteins to be produced for
the first time. Q. Has gene therapy cured any diseases so far?
A. Yes, but rarely. Gene therapy is a very young science that
has been used in patients for only about ten years. Thousands of
patients with cancer and infectious diseases, such as AIDS, have
been treated but have not been cured yet. While these are very
difficult disea