Bioengineering and Molecular Medicine
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The Bioengineering and Molecular Medicine group is a multidisciplinary laboratory research team based at The Children’s Hospital at Westmead. We have two decades of expertise with preclinical models in orthopaedic injury, brittle bone disease and neurofibromatosis.
Our team is unique in its multilevel integration of basic and preclinical programs, with a strong focus on clinical translation. We have a comprehensive track record of working with patient advocacy groups as well as biotech and medical industry partners.
Objectives
We conduct research to develop new therapeutics to treat children with musculoskeletal disorders and injuries. Our research aims to bring cures to children affected by brittle bone disease and neurofibromatosis as well as many other musculoskeletal gene disorders.
Impact
- Our research has led to improved clinical treatment for tibial pseudarthrosis, where amputation was previously a common outcome.
- A clinical trial published by us in 2022 showed that an over-the-counter nutraceutical supplement can improve muscle function in children with NF1.
- Our research has helped guide our understanding of combination drug therapies for brittle bone disease, including bisphosphonates and sclerostin antibody, and bisphosphonates and growth hormone. This information is currently used by clinicians to guide off-label prescription.
- We invented a new bone-binding antibiotic with the potential to reduce the rate of surgery-related infections.
- We were the first group to develop a bone targeted AAV vector and have subsequently shown its selectivity and efficiency in targeting bone cells.
Affiliations
