Michelle Ruhle 42049401
Gene Therapy
The article was about gene therapy which attempts to treat genetic diseases at the molecular level by restoring normal functions of proteins. The therapy hinges on finding an effective system to carry the correcting genes that will work in the long term. Researches often use viruses to deposit the genetic material but they are engineered so that they cannot reproduce and only deliver the desired DNA.
The researchers showed that the cells from patients with an ADA deficiency which leaves them defenceless against infection, could be corrected in a tissue culture. In this case they used a retrovirus to carry the ADA gene. They realised that while this process was not harmful it was not a successful treatment because not enough cells had the new DNA. To combat this they switched to using the patients white blood cells and this greatly increased the number of cells taking up the ADA gene. The patients have shown increases in ADA in their immune cells following gene therapy.
http://history.nih.gov/exhibits/genetics/sect4f.htm
