Investigating Drug Treatments For A Machado Joseph Disease Using Transgenic Zebrafish
Funder
National Health and Medical Research Council
Funding Amount
$443,425.00
Summary
Machado Joseph disease (MJD) is a hereditary neurodegenerative disease that causes problems with a patient’s co-ordination and movement, leading to paralysis and death. Although the disease affects patients throughout the world, it is most common within Aboriginal communities of Arnhem Land in the Northern Territory. This project seeks to identify a drug treatment for the disease by examining the effect of relevant drugs on zebrafish genetically modified to have the human gene that causes MJD.
Calpeptin, And Related Candidates, For The Treatment Of Machado Joseph Disease
Funder
National Health and Medical Research Council
Funding Amount
$888,040.00
Summary
Machado Joseph Disease (MJD) is a neurodegenerative disease that causes impaired movement and progressive paralysis, leading to patient death. MJD is inherited within families, including a high number of Indigenous families of northeast Arnhem Land. We have identified a possible treatment for MJD that has positive effects on a small animal model of the disease (zebrafish carrying the human MJD gene). We plan to test this treatment further with the aim of developing a treatment for MJD patients.
Defining FMR1 And SNRPN Epigenetic Signatures Associated With Neurodevelopmental Disorders
Funder
National Health and Medical Research Council
Funding Amount
$318,768.00
Summary
Fragile X Syndrome and imprinting disorders such as Prader-Willi Syndrome and Angelman Syndrome are characterised by variable penetrance for intellectual disability, motor delay and autism spectrum disorder. This project aims to investigate the prognostic value of using blood-based biomarker tests and sensitive neuroscience informed measures to predict risk and severity of neuropsychological problems in children affected by these disorders.