Research Home Michigan State University, Dr. Margaret Jones, M.D. Previous research in my laboratory suggested that enzyme replacement therapy (ERT) for the treatment of Sanfilippo D Syndrome in our goat animal model reduced substrate storage in some organs, but was not effective in the brain. We presume enzyme is unable to cross the protective blood-brain barrier. Our research group's recent attempts at therapy for Sanfilippo in our goat animal model have focused on bypassing this barrier by direct delivery of enzyme or cells into the brains of these animals. Injection of purified enzyme (from Dr. John Hopwood's laboratory) into the brains of two newborn goat kids was performed. While results have not been fully analyzed, correction of the storage was, unfortunately, not obvious. We also have continued with our efforts to transplant neuroprogenitor cells from mice into the brains of fetal goats (during a pre-immune period of development, to try to avoid immune issues common in transplants). It is hoped that these cells, or genetically modified versions of them, may be able to implant into the brains to provide corrective enzyme to affected cells. Results of these trials also are pending further analysis. The laboratory has been undergoing a period of transition as I make plans to relocate the research program to other institutions. The valuable Sanfilippo goats soon will be on their way to the University of Georgia to support the research of Dr. K. Paige Carmichael and Drs. Robert Yu and Stacey Kraemer. Dr. Stacey Kraemer, a long-time research associate in my laboratory, will be relocating soon to the Medical College of Georgia to continue Sanfilippo research with Dr. Robert Yu. Samples from the Sanfilippo goats are being sent to Dr. John Hopwood (Adelaide, South Australia), where they will be used for further Sanfilippo research. Collaborations with Drs. Hopwood, Yu, Carmichael, and Kraemer will continue. Cells and/or semen from the Sanfilippo goats also have been relocated to further benefit future research on Sanfilippo Syndrome. Inquiries can be made regarding their availability to Dr. Mark Haskins (University of Pennsylvania) and Dr. Esmail Zanjani (University of Nevada – Reno).