Projects

Assessment of Epigenetic Driven Circadian Rhythm Defects in Neurons from Individuals with PWS

Funding Summary Dr. Reiter is analyzing neurons, made from stem cells from individuals with PWS.  He has noted disruptions in the circadian rhythm of these cells (day/night cycle) that may reflect sleep problems in PWS.  His work will identify how the circadian rhythms are disrupted in PWS cells, and pave the way for identifying new drugs to false

Identification of Critical Periods for the Neurodevelopmental and Behavioral Effects of Oxytocin

Funding Summary 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 false

Understanding the Role of Microglia in the Prader-Willi Hypothalamus

Funding Summary Dr. Kurrasch is studying whether inflammation in the brain, mediated by special immune cells called microglia, might contribute to hyperphagia and obesity in PWS. Using a mouse model of PWS, she will examine microglia activity and explore whether eliminating microglia improves energy regulation. Funding provided by FPWR – Canada.

Newborn Screening for Prader-Willi and Angelman Syndromes

Funding Summary Universal newborn screening for PWS will ensure that all babies with PWS are diagnosed at birth. Dr. Godler has developed a sensitive, accurate and cost-effective DNA test for detection of PWS and Angelman syndrome using the bloodspots (“heelprick”) obtained in all newborns.  In the study, FPWR is collaborating with with the false

Cannabidivarin (CBDV) vs Placebo in Children with PWS

Funding Summary This grant will support a small clinical trial evaluating the impact of cannibidiol cannabidivarin (CBDV) on hunger and behavior.  CBDV is a compound similar to CBD, but with potential advantages over CBD for PWS. In this study, Dr. Hollander’s group will investigate a form of CBDV, a non-psychoactive drug that has false

Design and Implementation of Hypothalamus-Specific Exosomes to Restore SNORD116 Deletion in PWS

Funding Summary Dr. Lee’s group is exploring the use of a novel gene transfer vehicle, exosomes, to deliver missing portions of the PWS genes to the hypothalamus. In this pilot study, they seek to develop PWS-specific exosomes and test how well these can deliver genes to neurons and other cells.

Acceptance and Commitment Training (ACT) to Reduce Stress in Fathers of Adolescents with PWS

Funding Summary Families of those with PWS experience a great deal of stress, particularly during adolescence. In this study, we are funding the development of a new behavioral intervention, aimed at developing more effective coping skills in fathers of adolescents with PWS, as a means to improve overall family well being. Dr. Forster will work false

Guanfacine XR for Aggression and Self Injury in PWS A Double Blind Placebo Controlled Trial

Funding Summary Guanfacine XR (brand name Intuniv) is a medication for ADHD that improves impulse control. Dr. Singh has noted improvements in aggression and self injury in PWS patients in his practice when using this medication. In this study, he will perform a pragmatic clinical trial to evaluate the efficacy of guanfacine for treating these false

Generation of Non-Human Primate Models of PWS

Funding Summary This project takes the first steps towards developing a primate (macaque) model of PWS. Animal models of PWS are currently limited and are not able to replicate some important aspects of PWS, such as intellectual disability, as well as behavioral and social impairments. These aspects of PWS may be more effectively studied in a false

Chronic Stress, Cognition, and Food Cue Reactivity in PWS. A Magnetoencephalography Study

Funding Summary This study will combine advanced brain imaging technology with other assessments to examine how hormonal, cognitive, and psychological factors are interrelated in PWS. Results from this study will increase the understanding of how brain regions involved in food intake are related to appetite hormones, hair cortisol, and false

Cannabinoid-1 Receptor Blockade to Treat Hyperphagia, Obesity and Related Metabolic Disorders in PWS

Funding Summary This grant supports the development of a new drug to tackle hunger and obesity with PWS. Inversago, a newly started, specialized biotech company, proposes that its CB1 blockers would treat a wider spectrum of symptoms than anything presently in development for PWS. The drug targets the endocannabinoid pathway, which is known to be false

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