OXPHOS Upregulation To Preserve Vision In Leber's Hereditary Optic Neuropathy
Funder
National Health and Medical Research Council
Funding Amount
$496,874.00
Summary
Leber's Hereditary Optic Neuropathy (LHON) is a devastating blinding disease that preferentially affects young men. Sufferers have normal vision until teenage years or their twenties when a rapid loss of vision occurs that results in permanent blindness. It is caused by genetic changes in the mitochondrial DNA that we inherit from our mothers. The mitochondria are the cells' energy generators. We aim to use molecules similar to female hormones to boost energy as a new treatment to preserve visio ....Leber's Hereditary Optic Neuropathy (LHON) is a devastating blinding disease that preferentially affects young men. Sufferers have normal vision until teenage years or their twenties when a rapid loss of vision occurs that results in permanent blindness. It is caused by genetic changes in the mitochondrial DNA that we inherit from our mothers. The mitochondria are the cells' energy generators. We aim to use molecules similar to female hormones to boost energy as a new treatment to preserve vision in at-risk LHON individuals.Read moreRead less
Modelling Leber’s Hereditary Optic Neuropathy Using Human Induced Pluripotent Stem Cells
Funder
National Health and Medical Research Council
Funding Amount
$628,416.00
Summary
Leber’s Hereditary Optic Neuropathy (LHON) is a blinding disease that affects young males and is caused by the death of cells in the optic nerve. To better understand LHON, this project utilises induced pluripotent stem (iPS) cells for disease modelling. iPS cells will be generated from patients and turned into optic nerve cells, allowing us to study the diseased cells in the laboratory, providing a platform to screen for novel drugs to improve treatment options and fast-track drug development.