Treatment of Osteoporosis in Murine Rett Syndrome Models: Comparison of Zoledronic Acid vs. Teriparatide on Osteoblast Function, Gene Expression and Bone Mass

Principal Investigator: Jay Shapiro

This project developed from my practice of seeing patients with Rett Syndrome (RTT) and bone abnormalities at the Kennedy Krieger Institute.  The recognition of osteoporosis and the resulting fractures in this population led to a review of the Kennedy Krieger patients with Dr. Sakkubai Naidu, which was published in 2010 1. Studies related to deficient bone function in RTT were also initiated using the Bird mouse model, and was presented at the Meeting of the Americal Society for Bone and Mineral Research in 2007 2.

The significance of this investigation includes the following: 1) the potential for treating osteoporosis and decreasing fractures in RTT children and young adults, and 2)  the importance  of observations related to MeCP2 mutations which affect the growth and maturation of nerve tissue and may be affecting the osteoblast in the same manner leading to osteoporosis. We will focus on this as our research proceeds.