Synchrotron X-ray Assessment Of Airway Surface Physiology For Cystic Fibrosis
Funder
National Health and Medical Research Council
Funding Amount
$778,228.00
Summary
We seek a cure or long-lasting therapy for the fatal airway disease in cystic fibrosis. Disease is caused by a shallow and dehydrated airway surface liquid (ASL), allowing bacteria to infect the lung. We can introduce a corrective gene into mouse airways where it can be effective for over 1 yr, but no fast, accurate and non-invasive measurement exists to test if treatments are successful. We will develop methods using synchrotron light to directly measure ASL depth changes in live mouse airways.
Linkage Infrastructure, Equipment And Facilities - Grant ID: LE0882382
Funder
Australian Research Council
Funding Amount
$245,000.00
Summary
Biophysical Characterisation Facility. The protein analysis facility will have substantial benefits for basic science and biotechnology. It will create capacity for South Australian researchers to study proteins at the biophysical level. The facility will support research projects within the designated national research priority areas of 'Frontier technologies for building and transforming Australian industries' and 'Promoting and maintaining good health
ARC Centre of Excellence in Convergent Bio-Nano Science and Technology. The CoE in Convergent Bio-Nano Science &Technology comprises a multi-disciplinary team focused on research aiming to understand and control the interface of materials with biological systems. The Centre will exploit knowledge of the bio-nano interface to design materials that transport and deliver vaccines, drugs and gene therapy agents, and to design new diagnostic agents and devices. Nanomedicines are on the cusp of revol ....ARC Centre of Excellence in Convergent Bio-Nano Science and Technology. The CoE in Convergent Bio-Nano Science &Technology comprises a multi-disciplinary team focused on research aiming to understand and control the interface of materials with biological systems. The Centre will exploit knowledge of the bio-nano interface to design materials that transport and deliver vaccines, drugs and gene therapy agents, and to design new diagnostic agents and devices. Nanomedicines are on the cusp of revolutionizing diagnosis and therapy in many diseases. The CoE will be the focus of bio-nano research activity in Australia, uniting universities, research agencies, institutes and companies. The expected outcomes are better diagnostic and therapeutic tools designed via an enhanced understanding of the bio-nano-interface.Read moreRead less
Development of a gene delivery system to access neuronal cells. Understanding the pathways for gene delivery and efficient expression will result in new knowledge in the areas of biotechnology and cell biology. This project will result in significant new information on vesicular trafficking pathways in neurons. An effective gene delivery system will realise significant commercial potential for our partner organisations and economic benefit to other Australian industry. This project aligns direct ....Development of a gene delivery system to access neuronal cells. Understanding the pathways for gene delivery and efficient expression will result in new knowledge in the areas of biotechnology and cell biology. This project will result in significant new information on vesicular trafficking pathways in neurons. An effective gene delivery system will realise significant commercial potential for our partner organisations and economic benefit to other Australian industry. This project aligns directly with the National Research Priority of "Promoting and maintaining good health" with a specific benefit for patients that suffer mental and physical degeneration and for their families.Read moreRead less
Single Minded 2: Cross coupling or specificity within the bHLH/PAS transcription factor family? Understanding the mechanisms of action of SIM2 may lead to novel ideas towards drug development for diseases such as Down syndrome and cancer. The SIM2 protein can interfere with activity of the related Hypoxia Inducible Factor (HIF), a protein important in stress response and recovery from stroke. Understanding the molecular basis of this interference could aid current strategies being used to manipu ....Single Minded 2: Cross coupling or specificity within the bHLH/PAS transcription factor family? Understanding the mechanisms of action of SIM2 may lead to novel ideas towards drug development for diseases such as Down syndrome and cancer. The SIM2 protein can interfere with activity of the related Hypoxia Inducible Factor (HIF), a protein important in stress response and recovery from stroke. Understanding the molecular basis of this interference could aid current strategies being used to manipulate HIF for pharmaceutical benefit.Read moreRead less
How does timing affect mammalian brain development and evolution? This project aims to generate fundamental knowledge on the origin of diversity in mammalian brain circuits by studying development of marsupials and rodents. The expected outcome is to elucidate how differences in the timing, rate and sequence of development of gene expression, cell differentiation and circuit formation can relate to the origin of key evolutionary innovations in the mammalian brain. The significance of understandi ....How does timing affect mammalian brain development and evolution? This project aims to generate fundamental knowledge on the origin of diversity in mammalian brain circuits by studying development of marsupials and rodents. The expected outcome is to elucidate how differences in the timing, rate and sequence of development of gene expression, cell differentiation and circuit formation can relate to the origin of key evolutionary innovations in the mammalian brain. The significance of understanding the dynamics of developmental systems that shape complex brain traits includes establishing new developmental paradigms in evolutionary theory, generating new tools to investigate and manipulate brain gene expression in vivo, and the potential discovery of the causes of neurodevelopmental dysfunction.Read moreRead less
Many Ys in monotremes: multiple sex chromosomes and sex determination in platypus and echidna. Platypus and Echidna are Australian icons, even featuring on our coins. Their unusual biology and ancient relationship to humans make them unique for understanding the mammalian genome, as demonstrated by our recent discovery of ten sex chromosomes in platypus that link mammal and bird sex. Still little is known about their genome, embryo development and how sex is determined. We will identify new gene ....Many Ys in monotremes: multiple sex chromosomes and sex determination in platypus and echidna. Platypus and Echidna are Australian icons, even featuring on our coins. Their unusual biology and ancient relationship to humans make them unique for understanding the mammalian genome, as demonstrated by our recent discovery of ten sex chromosomes in platypus that link mammal and bird sex. Still little is known about their genome, embryo development and how sex is determined. We will identify new genes on all ten sex chromosomes and investigate how they determine sex. The set up of an Australian Monotreme Resource Centre will be crucial for this research and attract worldwide high profile collaborations. We will answer important general questions in monotreme biology and contribute to our understanding of sexual abnormalities in humans.Read moreRead less
Understanding the molecular mechanisms of intellectual disability. Intellectual disability is frequent in the population, with one in every fifty people in the world directly affected. This project will improve our understanding of the correct development and function of the brain required for cognition by investigating specific roles and regulation of key molecules involved.
The Role Of UPF3B And Nonsense Mediated MRNA Decay Surveillance In The Pathology Of Intellectual Disability.
Funder
National Health and Medical Research Council
Funding Amount
$789,954.00
Summary
Proper functioning of the nonsense mediated mRNA decay (NMD or 'mRNA police') is crucial for any cell to ensure normal development and function. When NMD is compromised the outcome is learning and memory problems, autism or schizophrenia. Under this project we study malfunctioning NMD using stem and neuronal cells derived from patients' skin cells. Some of the affected genes might be considered for therapeutic interventions. NMD is relevant to 1000s of human disorders and as such it is of fundam ....Proper functioning of the nonsense mediated mRNA decay (NMD or 'mRNA police') is crucial for any cell to ensure normal development and function. When NMD is compromised the outcome is learning and memory problems, autism or schizophrenia. Under this project we study malfunctioning NMD using stem and neuronal cells derived from patients' skin cells. Some of the affected genes might be considered for therapeutic interventions. NMD is relevant to 1000s of human disorders and as such it is of fundamental importance.Read moreRead less
About one in eight known genetic disorders involve DNA alteration that activates a cellular quality control mechanism that disables the affected gene. This mechanism is more efficient in some individuals than others. It can influence disease outcomes and severity. We will engineer and apply tools and models to measure and manipulate this crucial cellular mechanism. This will allow us to predict disease severity as well as to intervene where a manipulation of this mechanism will be beneficial.