RNA Interference And Retigabine Therapy Protect Against Hereditary Hearing Loss
Funder
National Health and Medical Research Council
Funding Amount
$370,522.00
Summary
The preservation of hearing function is central to the treatment of individuals who are genetically predisposed to hearing loss. At present only synthetic hearing aids and cochlear implants can provide functional improvement, albeit sub-optimal. The studies described here will seek to prevent hearing loss by reducing the damaging effects of defective genes. Gene therapies that reduce the effect of these defective genes and a drug that enhances the activity of functional genes will be developed.
Use of Gradipore technology to develop novel methods for the preparation and segregation of mammalian spermatozoa. The purpose of this project is to harness the expertise available within an Australian biotechnology company, Gradipore, to develop novel methods for the preparation of mammalian spermatozoa and the segregation of these cells into X-and Y- bearing populations. This technology will find application in: (1)clinical andrology, where rapid, safe protocols for the preparation and segrega ....Use of Gradipore technology to develop novel methods for the preparation and segregation of mammalian spermatozoa. The purpose of this project is to harness the expertise available within an Australian biotechnology company, Gradipore, to develop novel methods for the preparation of mammalian spermatozoa and the segregation of these cells into X-and Y- bearing populations. This technology will find application in: (1)clinical andrology, where rapid, safe protocols for the preparation and segregation of human spermatozoa are being actively sought in the context of assisted conception and the management of sex-linked genetic diseases and (2) agriculture, particularly the cattle industry, where a capacity to predetermine the sex of the offspring would be extremely valuable.Read moreRead less
Cell-targeted Gene Delivery Into Human Haematopoietic Stem Cells For The Treatment Of Thalassaemia
Funder
National Health and Medical Research Council
Funding Amount
$171,208.00
Summary
Thalassaemia is the most common inherited single gene disorder affecting haemoglobin synthesis in red blood cells. It mainly affects people of Mediterranean, Middle Eastern, African, South East Asian, Chinese, and Indian origin. However, large numbers of thalassaemia patients are found nowadays in Australia and other developed countries, due to large population movements in the twentieth century. Approximately 300,000 severely affected children are born each year with thalassaemia and various ot ....Thalassaemia is the most common inherited single gene disorder affecting haemoglobin synthesis in red blood cells. It mainly affects people of Mediterranean, Middle Eastern, African, South East Asian, Chinese, and Indian origin. However, large numbers of thalassaemia patients are found nowadays in Australia and other developed countries, due to large population movements in the twentieth century. Approximately 300,000 severely affected children are born each year with thalassaemia and various other abnormalities of haemoglobin synthesis. If untreated, most thalassaemia patients will die within the first few years of life. The vast majority of thalassaemia patients depend on regular blood transfusions every two to three weeks, and on nightly infusions of an iron chelator (a drug for removing excess iron from the blood). These procedures place considerable burden on thalassaemia patients, their families and society, and expose them to blood transmitted infections. The only curative treatment for thalassaemia is bone marrow transplantation from a matching donor. However, the vast majority of patients do not have matching donors and thus the only prospect for them to receive such therapy is to replace in their bone marrow cells a copy of the normal set of genes for the synthesis of haemoglobin. The studies in this proposal are therefore designed to test gene therapy protocols on bone marrow stem cells derived from thalassaemia patients. A normal set of globin genes will be delivered to the bone marrow stem cells via non-viral delivery systems and examined for function in an immunodeficient mouse strain that can accept human bone marrow. This research may enable bone marrow transplantation to be applied for the therapy of most patients with thalassaemia, while it may also have a major impact on therapeutic approaches for other haematological anomalies.Read moreRead less
Customized IPS Cell Therapy For Recessive Monogenic Retinal Degenerative Disease
Funder
National Health and Medical Research Council
Funding Amount
$350,714.00
Summary
The focus of this study is to develop a personalised treatment for certain types of retinal degenerative disease (RDD). Stem cells will be generated from the skin cells obtained from an individual with RDD. Gene therapy will then be applied to correct the underlying disease-causing mutation in the patient cells. The repaired cells will be used to generate retinal cells, which will subsequently be tested in naturally occurring RDD rodent models to determine if they have any beneficial effects.
Gene therapy to enhance auditory prosthesis performance for cochlear implants. The cochlear implant is the most effective neural prosthesis, restoring hearing to the deaf. The research aims to develop a new type of implant compatible with delivery of therapeutic genes to the cells lining the cochlea. Gene therapy DNA constructs will be developed that will enhance neural survival and growth, improving cochlear implant performance. The research will provide advances in understanding how to tran ....Gene therapy to enhance auditory prosthesis performance for cochlear implants. The cochlear implant is the most effective neural prosthesis, restoring hearing to the deaf. The research aims to develop a new type of implant compatible with delivery of therapeutic genes to the cells lining the cochlea. Gene therapy DNA constructs will be developed that will enhance neural survival and growth, improving cochlear implant performance. The research will provide advances in understanding how to transfer genes into cochlear tissue, as well as development of gene cassettes for effective neural repair. The work will advance the field of bionics, an area where Australia is establishing international preeminence.Read moreRead less