Miller will discuss that question and the problems facing gene therapy in a lecture on Friday, Feb. 6, from noon to 1 p.m. in room T-625 of the Health Sciences Center. Scientists spend too much time touting preliminary findings that have been blown out of proportion, Miller says. What is needed for gene therapy to move forward is vigorous attention to fundamental problems, he added. In gene therapy, researchers typically use viruses to transfer DNA to cells with genetic defects. Retroviruses promote stable integration of their genes into the DNA of the target cell, allowing persistent expression of therapeutic genes carried by the virus.
Specific rules regulate how the viruses recognize their target cells, moreover.
The receptor acts as a beacon of sorts that the virus locks onto to navigate into the cell. Patients with SCID have very low numbers of circulating T cells and experience recurrent severe infections. The researchers used a retrovirus to pass along DNA to the children that corrected the mutation. After treatment, the children's T cell levels returned to nearly normal levels. Today, seven years later, the T cells in one of the children are still expressing the healthy gene, he said.