Cures for cancer, Parkinson’s disease, baldness…a vaccine against HIV.. …stopping heritable genetic disorders before they start…This is the future of gene therapy—and the present of the genetic research.  Consider the possibilities….
 

 Adenosine deaminase, or ADA, deficiency is cause by a defect in a single gene that affects fewer than 10 children each year in North America.  Victims cannot produce the enzyme ADA that is necessary for a healthy immune system.  Without ADA, a substance that is toxic to T and B lymphocytes builds up in the blood, and the immune system is severely compromised.  ADA victims die early in childhood.

 In 1986, Michael Blaese, French Anderson, and their colleagues at the National Institutes of Health made copies of the non-defective ADA gene and used a retroviral vector to insert the gene into cultured B and T lymphocytes from children with ADA deficiency.  The tactic was a success; the gene made enough ADA to suggest that it would deliver a usable dosage to a patient.  Should this technique prove valid in human trials, families across North America would be profoundly grateful—and so would the company that owned a patent on that gene. (Culliton, “ADA: A Prime Candidate”)
 

 A significant percentage of emphysema cases are actually caused by a genetic defect, the absence of the gene that produces a protein called alpha-1 antirypsin.  Alpha-1 acts as a shield to protect the lungs from being eaten away by neutrophil elastase.  When it’s absent, victims are vulnerable to emphysema.  Few victims live past 60.  Ronald G. Crystal wants to insert the alpha-1 gene into T-lymphocytes which would be put into an aerosol spray.  Victims of emphysema could then spray the lymphocytes directly into their airways, where they would produce sufficient quantities of alpha-1 to protect the lungs from further damage.  (Culliton, “A Genetic Shield to Prevent Ephysema?”)
 

 Recently, researchers have found a molecule on immune cells—the CCR5 receptor—that allows the virus that causes AIDS into the cell.  This discovery could lead to new drugs that block HIV infections—and significant profits for whoever holds the patent on this gene.  Sorry, customers, but Human Genome Sciences has applied for this one.  Don’t be left out of the loop again.  Get your patents now!  (Marshall, “HIV Experts vs. Sequencers in Patent Race”)