Much of the work to develop a gene therapy for LAD was done by Tom Bauer, a researcher at the VA who helped spearhead the clinical trial. Although Hickstein originally cloned the missing gene for LAD, it was Bauer who spent the past seven years developing the protocol using gene therapy to treat the disease. The genetically altered stem cells were then reintroduced into Lucas' bloodstream. The technique is based on the ability of a special class of viruses, called retroviruses, to insert their own genetic material into the genomes of the cells they infect. For gene therapy, the retrovirus has been modified so instead of transferring its own disease-causing genes, it transfers a piece of foreign DNA, in this case a correct copy of the gene that will tell Lucas' body how to make the missing protein it needs to fight infections.
Initial reports are positive.
One month after receiving her genetically altered cells, her body was showing signs that the treatment was beginning to work. In the first patient's case, the number of white blood cells responding to the scraping site had more than doubled, compared with a similar test done before treatment. Patients don't need 100 percent restoration of gene function to fight infection. You don't need much. It will be several months before scientists have a good read on whether gene therapy has worked for Lucas.