Correction Of Friedreich Ataxia Induced Pluripotent Stem Cells By Non-viral Gene Therapy
Funder
National Health and Medical Research Council
Funding Amount
$63,270.00
Summary
Friedreich ataxia (FRDA) is an inherited progressive disorder of the nervous system and heart. Stem cell therapy has the potential to repair or replace damaged tissues and restore organ function in FRDA patients. The defect inherent in stem cells obtained from FRDA patients will be corrected by a gene therapy approach that will restore normal FRDA gene expression and addresses major safety concerns for the clinical use of corrected stem cells in transplantation medicine.
A Novel Approach To Cytokine Blockade For The Treatment Of Systemic Lupus Erythematosus
Funder
National Health and Medical Research Council
Funding Amount
$137,700.00
Summary
Lupus is a disease of the immune system which can cause inflammation and damage to many organs and even death, often affecting young people in their prime of life. Current treatments are limited and often have undesirable side effects. Certain cells and molecules are thought to be responsible for causing lupus. This project will examine the effect of blocking one of these molecules as a means of treating lupus. It will also use gene and protein analysis of samples from lupus patients to see if r ....Lupus is a disease of the immune system which can cause inflammation and damage to many organs and even death, often affecting young people in their prime of life. Current treatments are limited and often have undesirable side effects. Certain cells and molecules are thought to be responsible for causing lupus. This project will examine the effect of blocking one of these molecules as a means of treating lupus. It will also use gene and protein analysis of samples from lupus patients to see if response to treatments can be predicted.Read moreRead less
Gene Therapy And Bone Marrow Transplantation For Treatment Of Experimental Autoimmune Uveitis
Funder
National Health and Medical Research Council
Funding Amount
$78,445.00
Summary
Bone marrow stem cells modified to express self antigen is proposed as a treatment for experimental autoimmune uveitis. A major goal of this research is to investigate the impact and possible mechanism of gene therapy on the development and progression of autoimmune disease in the eye to prevent or reduce the severity of disease.
Antibody Targeted Virus Particles For A Gene Therapy Approach To Inhibiting Atheroschlerosis Development
Funder
National Health and Medical Research Council
Funding Amount
$95,313.00
Summary
I am a Biotechnologist and my research looks into ways of preventing Atherosclerosis. Atherosclerosis is the build up of plaques in artery walls, and is the major precursor condition to stroke and myocardial infarction (heart attack). My project focuses on a preventative gene therapy which will be delivered specifically to early stage plaques. The gene will inhibit one of the earliest developmental stages of atherosclerosis: recruitment of immune cells to these sites, and so prevent their growth ....I am a Biotechnologist and my research looks into ways of preventing Atherosclerosis. Atherosclerosis is the build up of plaques in artery walls, and is the major precursor condition to stroke and myocardial infarction (heart attack). My project focuses on a preventative gene therapy which will be delivered specifically to early stage plaques. The gene will inhibit one of the earliest developmental stages of atherosclerosis: recruitment of immune cells to these sites, and so prevent their growth.Read moreRead less
Bowel cancer is the 2nd most common cause of cancer death in Australia. Rectal cancer represents 40% of these, and is more common in the elderly who are frequently unable to tolerate chemoradiation therapy. The Mutated in Colorectal Cancer gene (MCC) could become a predictor to chemoradiotherapy in up to 30% of these patients. A defective MCC in tumours can predict a good response to this treatment. Our project will potentially identify patients that are more sensitive to chemoradiotherapy and l ....Bowel cancer is the 2nd most common cause of cancer death in Australia. Rectal cancer represents 40% of these, and is more common in the elderly who are frequently unable to tolerate chemoradiation therapy. The Mutated in Colorectal Cancer gene (MCC) could become a predictor to chemoradiotherapy in up to 30% of these patients. A defective MCC in tumours can predict a good response to this treatment. Our project will potentially identify patients that are more sensitive to chemoradiotherapy and lead to a personalized treatment of rectal cancer.Read moreRead less
A New Therapeutic Monoclonal Antibody Targeting CD302 In Acute Myeloid Leukaemia
Funder
National Health and Medical Research Council
Funding Amount
$124,676.00
Summary
This project will develop a new antibody treatment for Acute Myeloid Leukaemia. Antibody treatments help the body to attack the leukaemia using its immune system. The prognosis of this leukaemia is poor. Our current treatments use high dose chemotherapy and sometimes a stem cell transplant. Many patients cannot have the current therapy due to their age or other medical problems. A new antibody therapy may be used on its own or with other therapies to help more patients achieve remission.
Successful establishment of pregnancy requires a viable embryo and a receptive uterus. The contribution that reduced uterine receptivity makes to human infertility is unknown. IVF patients with implantation failure will be investigated using different approaches to identify gene pathways that are altered in reduced uterine receptivity. This study could lead to a clinical test for uterine receptivity and help to improve fertility treatment options for women with repeated implantation failure.
Mechanisms Of Gender Differences In Genetic Aortopathy
Funder
National Health and Medical Research Council
Funding Amount
$122,686.00
Summary
This project will investigate the molecular mechanisms that underly the gender differences in phenotypic expression in young adults with genetic aortopathy.
The Use Of Real-World Evidence To Support Regulatory And Reimbursement Decisions
Funder
National Health and Medical Research Council
Funding Amount
$91,538.00
Summary
Traditionally, medicines are studied extensively in clinical trials before they are widely available. More recently, some medicines have been allowed to enter the market without complete data on their benefits and risks. This means that these issues can only be studied once a medicine is on the market and used in routine clinical practice; this is referred to as real-world evidence. This research evaluates if this evidence is sufficient to prove that a medicine is safe and that it works
Lung cancer is the commonest cause of cancer death worldwide. There is growing interest in the genetic causes of lung cancer. The overall aim of this research is to gain a better understanding of the steps in the genetic pathway of lung cancer spread. This knowledge is essential in the development of new targeted therapies and improvement in lung cancer prognosis.