Charged Lipophilic Dendrimers: Delivery Of Oligonucleotides With Therapeutic Potential
Funder
National Health and Medical Research Council
Funding Amount
$422,036.00
Summary
Choroidal neovascularisation, which is the most severe form of Age Related Macular Degeneration (AMD) is the major cause of blindness in the developed world. The disease usually affects people above the age of 75-80. With an ageing population, reaching 3.5 million (over 65) in Australia by year of 2020, AMD is quickly becoming a significant socio-economic problem. Gene therapy could be a cure for the above disease. Currently, there are large numbers of antisense oligonucleotides that have the po ....Choroidal neovascularisation, which is the most severe form of Age Related Macular Degeneration (AMD) is the major cause of blindness in the developed world. The disease usually affects people above the age of 75-80. With an ageing population, reaching 3.5 million (over 65) in Australia by year of 2020, AMD is quickly becoming a significant socio-economic problem. Gene therapy could be a cure for the above disease. Currently, there are large numbers of antisense oligonucleotides that have the potential to be developed as new medicines. However, a lack of absorption-cellular uptake and poor in vivo stability are major hurdles that must first be overcome, before any of these compounds will reach the clinic. Synthetic DNA delivery agents are of interest for gene therapy as an alternative to viral vectors, since they carry potentially fewer risks in terms of immuneresponse and propagation. Gene transfer with synthetic compounds is a growing field of research and the largest family of such agents is based on positively charged lipids which are able to self-associate and to form complexes (salts) with DNA conferring a compacted state on the plasmid. Our project will address these major issues through a highly novel strategy involving ion pair formation of lipophilic dendrimer (tree-like compounds with positive charges on the surface) constructs. This multidisciplinary approach has the potential to develop and test new DNA-dendrimer complexes and test them in a well established animal model for neovascularisation. Successful completion of this project might offer a potential therapy for choroidal neovascularisation, with a good chance of entering into human clinical trials by year 2004.Read moreRead less
Minimally-invasive Gene Delivery Of A Novel Inhibitor Of Retinal Angiogenesis
Funder
National Health and Medical Research Council
Funding Amount
$883,883.00
Summary
Excessive growth of blood vessels in the eye causes vision loss and can only be treated with lasers or painful and frequent injections into the eye. Vasostatin is a specific inhibitor of angiogenesis and a promising agent for the management of ocular neovascularisation. We will provide pre-clinical evidence that gene delivery of vasostatin-like peptides is an effective therapeutic strategy and it has potential to revolutionize the current ophthalmic care of age-related macular degeneration.
The Role Of Estrogen Signalling In The Development And Progress Of Neovascularisation In Macular Degeneration
Funder
National Health and Medical Research Council
Funding Amount
$318,768.00
Summary
Age-related macular degeneration is a common eye disease. In the advanced stages of the disease, abnormal and leaky blood vessels form, causing permanent and severe vision loss. A novel treatment is the application of the sex hormone, estrogen, which could halt abnormal blood vessel growth in the eye. This project aims to confirm the protective effects of estrogen on eye health and whether mutations in estrogen-related genes alter the risk of vision loss due to abnormal blood vessel growth.
A Novel Treatment For Ameliorating Retinal Vascular Disease
Funder
National Health and Medical Research Council
Funding Amount
$366,685.00
Summary
Retinal vascular disease is a leading cause of blindness and is currently treated by laser photocoagulation surgery. Although successful, this treatment is associated with serious side effects. Recently, Ellex Pty has developed a novel laser called the 2RT laser that is likely to be effective without the accompanying side effects. This study will allow examine the effect of the 2RT laser in animal models of retinal vascular disease so as to complete preclinical development of this laser.
Glial-neuronal-vascular Interactions In A Novel Transgenic Model Of Muller Cell Dysfunction
Funder
National Health and Medical Research Council
Funding Amount
$626,585.00
Summary
Muller cell disfunction is a feature shared by many retinal diseases. This project aims to study the contribution of Muller cell dysfunction to retinal neuronal damage and blood-retinal barrier breakdown in a novel transgenic model we recently generated. Results of this study will also be of interest to scientists and clinicians seeking to understand better and treat diseases of the central nervous system in general.