A5011989403- Virus-based gene therapy research
- CRISPR and Genetic Engineering
- RNA Interference and Gene Delivery
- Sexual Differentiation and Disorders
- Immune Cell Function and Interaction
- Metabolism and Genetic Disorders
- Adrenal Hormones and Disorders
- Renal Transplantation Outcomes and Treatments
- Animal Genetics and Reproduction
- T-cell and B-cell Immunology
- Epigenetics and DNA Methylation
- Genetics and Neurodevelopmental Disorders
Sydney Children’s Hospitals Network
2023-2024
Children's Medical Research Institute
2023-2024
The University of Sydney
2023-2024
University of Groningen
1998
Despite the availability of life-saving corticosteroids for 70 years, treatment adrenal insufficiency is not able to recapitulate physiological diurnal cortisol secretion and results in numerous complications. Gene therapy an attractive possibility monogenic adrenocortical disorders such as congenital hyperplasia; however, requires further development gene transfer/editing technologies knowledge target progenitor cell populations. Vectors based on adeno-associated virus are leading system direct
Congenital adrenal hyperplasia due to 21-hydroxylase deficiency leads high morbidity and mortality, despite the availability of life-saving corticosteroid replacement therapy. Gene therapy represents a promising potential treatment for monogenic disorders such as congenital hyperplasia, overcoming limitations approaches. Adeno-associated viral vectors are currently leading vector direct in vivo gene delivery. However, physiological properties gland limit application adeno-associated...
Conventional adeno-associated viral (AAV) vectors, while highly effective in quiescent cells such as hepatocytes the adult liver, confer less durable transgene expression proliferating owing to episome loss. Sustained therapeutic success is therefore likely liver disorders requiring early intervention. We have previously developed a hybrid, dual virion approach, recombinant AAV (rAAV)/piggyBac transposon system capable of achieving stable gene transfer at levels many fold above conventional...
Realization of the immense therapeutic potential epigenetic editing requires development clinically predictive model systems that faithfully recapitulate relevant aspects target disease pathophysiology. In female patients with ornithine transcarbamylase (OTC) deficiency, an X-linked condition, skewed inactivation X chromosome carrying wild-type OTC allele is associated increased severity. The majority affected can be managed medically, but a proportion require liver transplantation. With...