Projects

Under the guidance of our Scientific Advisory Board through a carefully managed grants process, FPWR selects research projects based on the collaborative input of researchers and parents, choosing projects that are both scientifically meritorious and highly relevant for individuals with PWS and their families.

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The Role of the Placenta in PWS: Mapping the Expression of PWS Genes

Funded Year: 2020

Dr. Isles and other researchers have shown that abnormal placental function can have profound consequences for brain and behavioral development in the offspring, and that abnormal signaling from the fetal placenta can also have consequences for maternal brain and behavior, which in turn may impact offspring neurodevelopment. This project examines...

Gene Therapy of Obesity in Prader-Willi Syndrome by an Autoregulatory BDNF Vector

Funded Year: 2020

Hyperphagia and the associated metabolic dysregulation is one of the greatest challenges that individuals with PWS and their families face on a daily basis. Dr. Cao has developed a gene therapy that targets the metabolic roots of PWS within the brain’s center for energy regulation. Their group has developed an approach using a single dose of a...

Precise epigenome editing as a novel therapeutic opportunity for Prader-Willi syndrome

Funded Year: 2020

Dr. Mussolino and his team will use a novel approach to activate the maternal genes in the PWS regions. They are developing ‘designer epigenome modifiers’ (A-DEMs), to target key elements of the PWS-critical region on chromosome 15. This approach may allow more specific activation of genes in the PWS region of chromosome 15 for genetic therapy...

Identification of Critical Periods for the Neurodevelopmental and Behavioral Effects of Oxytocin (Year 2)

Funded Year: 2020

The goal of the second year of this research project is to determine, using a preclinical mouse model of PWS, when do the maximal health and biological effects of oxytocin occur (birth, infancy, puberty, or adult life). The study also examines neurological mechanisms by which oxytocin treatment exerts its effects on feeding and behavior in PWS....

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