Self-destructing CRISPR-constructs For Targeted Genome Editing In The Retina.
Funder
National Health and Medical Research Council
Funding Amount
$679,926.00
Summary
Despite the identification of specific mutations causing many inherited retinal dystrophies, all of these conditions are currently untreatable. We have established gene-editing techniques and have developed a novel mouse model, which will serve as a robust platform for testing different techniques of gene editing in the retina. No other group in the world is known to be using this platform for gene editing and our work will expedite the clinical translation of this technology.
Editing Beneficial Single Nucleotide Polymorphisms Into The Genome: A New Approach To Genetic Disease
Funder
National Health and Medical Research Council
Funding Amount
$646,979.00
Summary
?-thalassaemia and sickle cell anaemia are red blood cell diseases caused by deficiencies in adult ?-globin, a component of oxygen-carrying haemoglobin. Interestingly there is a rare group of patients whose symptoms are reduced by naturally occurring mutations that upregulate another globin gene to compensate for defective ?-globin. We are investigating how these beneficial mutations elevate globin levels with a view to engineering them into cells of affected patients to treat these disorders.
The Role Of Alpha-haemoglobin Stabilising Protein In Haemoglobin Production And As A Therapeutic For Thalassaemia.
Funder
National Health and Medical Research Council
Funding Amount
$320,936.00
Summary
Thalassaemias are the most common hereditary diseases effecting the production of red blood cells. The underlying cause of disease is a failure to produce normal quantities of haemoglobin (Hb; the essential oxygen-carrying molecule in blood), resulting in severe anaemia. We have discovered a new protein with an important role in Hb production. We will elucidate the function of this protein in red blood cells and investigate novel treatments for thalssaemia disease.
Designer DNA-binding Proteins Targeting Methylated DNA For Research And Therapeutic Purposes
Funder
National Health and Medical Research Council
Funding Amount
$583,444.00
Summary
A number of human genes function to suppress the onset or progression of cancer. In cancer sufferers, these genes are often switched off. The aim of this project is to engineer designer protein molecules that will be able to switch these tumor suppressor genes on again in a selective manner. Because the switching off of tumor suppressor genes is common to all forms of cancer, the new technology created in this work will potentially benefit patients suffering from any of a wide range of cancers.
Controlling the rate of transcription and translation of Rubisco transgenes effectively in higher-plant plastids. Genetic transformation of the circular genome of the plastids provides a containable means for modifying plant growth by manipulating photosynthesis. Although the transformation mechanism is precise, predicting the level of foreign gene expression is difficult because the amounts of messenger RNA and protein produced by foreign genes in plastids varies widely, even when the protein a ....Controlling the rate of transcription and translation of Rubisco transgenes effectively in higher-plant plastids. Genetic transformation of the circular genome of the plastids provides a containable means for modifying plant growth by manipulating photosynthesis. Although the transformation mechanism is precise, predicting the level of foreign gene expression is difficult because the amounts of messenger RNA and protein produced by foreign genes in plastids varies widely, even when the protein assembles without difficulty. This project will devise strategies for controlling this variability that will facilitate attempts to exploit plastid transformation for transplanting better versions of the photosynthetic CO2-fixing enzyme, Rubisco, into plants to improve their growth efficiency in terms of water, fertiliser and light use.Read moreRead less
Defining New Building Blocks for the Construction of Artificial Genetic Circuits. By characterising the components of a natural genetic switch, we will make available a set of well defined genetic building blocks for construction of rationally designed biological circuits. The ability to build such circuits would have significant economic benefit in areas such as metabolic engineering, to improve the efficiency of production of natural compounds from micro-organisms, and in biomedicine, for the ....Defining New Building Blocks for the Construction of Artificial Genetic Circuits. By characterising the components of a natural genetic switch, we will make available a set of well defined genetic building blocks for construction of rationally designed biological circuits. The ability to build such circuits would have significant economic benefit in areas such as metabolic engineering, to improve the efficiency of production of natural compounds from micro-organisms, and in biomedicine, for the controlled release of therapeutic compounds. The involvement of Honours and Ph.D students in this project will expose the next generation of Australian scientists to this emerging discipline. International collaboration leading to publications in high impact scientific journals will enhance Australia's scientific reputation.Read moreRead less