This grant supports a new collaboration between two scientists with complimentary expertise. Drs. Sebastian Bouret and Francoise Muscatelli will work to define critical periods for oxytocin use in PWS models, and optimize this therapeutic approach. Mouse models will be used to define the critical period during which oxytocin might provide the maximum biological effect and impact. The goal is to develop the optimal therapeutic strategy for oxytocin in PWS.
Theresa Strong, Director of Research Programs, shares details on this project in this short video clip.
Watch the full webinar describing the 11 research projects funded in this grant cycle here.
Prader-Willi syndrome (PWS) is a genetic disorder characterized by an insatiable appetite and a variety of neurological and behavioral dysregulation. The hormone oxytocin is currently a promising topic in PWS research and treatment. Clinical trials have shown that oxytocin treatment can have lasting effects on appetite drive, social interactions and anxiety in patients with PWS, particularly if treatment is done in babies and infants. However, it is less clear whether such long-term beneficial health effects of oxytocin treatment can be seen in adult patients. We know that many key biological processes are established during the perinatal period —that time just prior to and soon after birth – and that alterations in biological systems can be reversed more persistently if intervention occurs during early life. The goal of this research project is to determine, using a pre-clinical mouse model for PWS, when do the maximal health and biological effects of oxytocin occur (birth, infancy, puberty, or adult life). It is also to better understand the neurological mechanisms by which oxytocin treatment exerts its effects on feeding and behavior in PWS. This research project promise to help developing optimal therapeutic strategies to ameliorate and hopefully permanently reverse feeding, neurological, and behavioral symptoms in PWS.
Sebastien Bouret and Francoise Muscatelli
Children's Hospital of Los Angeles and INSERM, France