Autoimmune Gene Therapeutics Group
The Autoimmune Gene Therapeutics Group is the laboratory-based research arm pf the Department of Rheumatology at Westmead Hospital and is embedded within a broader translational research environment spanning immunology, therapeutic development, drug discovery and clinical research in autoimmune disease. Research in the laboratory spans a portfolio of projects aligned with the group’s two core research pillars. Within autoimmune disease, the group is developing genetic medicines that converts the liver into a biofactory for therapeutic protein output to control inflammation, with a particular focus on antibody-mediated and complement-driven kidney disease. These programs seek to establish new pharmaco-genetic approaches that move beyond conventional immunosuppression by targeting the underlying mechanisms of immune dysfunction. In parallel, the laboratory leads studies investigating the immunobiology of adeno-associated virus (AAV) gene therapy, including the role of pre-existing antibodies, the structural basis of anti-capsid immune responses, and the development of next-generation vectors capable of overcoming naturally acquired immunity. Together, these projects combine fundamental immunology with vector engineering to improve access to genetic medicines and accelerate the development of transformative therapies for patients with chronic immune-mediated disease. Through its integration with the Department of Rheumatology at Westmead Hospital, the Group also provides opportunities for Advanced Trainees, PhD candidates and clinician scientists to undertake translational research spanning laboratory discovery and clinical application.
Recent publications
A novel fusion protein reduces kidney complement in experimental C3 glomerulopathy.
March 2026
Pancreatic Trans-differentiation of NOD Mouse Livers Prevented Development of Hyperglycemia
Gene Therapy enhances deoxyribonuclease I treatment in anti-myeloperoxidase glomerulonephritis
July 2025
Structural characterization of antibody-responses following Zolgensma treatment for AAV capsid engineering to expand patient cohorts
Structural and functional characterization of capsid binding by anti-AAV9 monoclonal antibodies from infants after SMA gene therapy.
Altered Hippocampal and Striatal Expression of Endothelial Markers and VIP/PACAP Neuropeptides in a Mouse Model of Systemic Lupus Erythematosus
August 2026
Performance of Cardiotropic rAAV Vectors Is Dependent on Production Method
Conversion of the liver into a biofactory for DNase I using AAV vector gene transfer reduces neutrophil extracellular traps in a model of Systemic Lupus Erythematosus
Adeno-associated virus vector gene delivery elevates Factor I levels and down-regulates the complement alternative pathway in vivo
Gain-of-function factor H-related 5 protein impairs glomerular complement regulation resulting in kidney damage
March 2021
Restoring the natural tropism of AAV2 vectors for human liver
Efficient editing of OTC-deficient patient-derived primary human hepatocytes
Use of a Hybrid Adeno-Associated Viral Vector Transposon System to Deliver the Insulin Gene to Diabetic NOD Mice
A user’s guide to the inverted terminal repeats of adeno-associated virus
Direct recognition of hepatocyte-expressed MHC class I alloantigens is required for tolerance induction
AAV-mediated gene delivery of the calreticulin anti-angiogenic domain inhibits ocular neovascularization
A novel liver-specific enhancer-promoter element in the wild-type AAV2 genome provides a mechanism for AAV participation in the pathogenesis of human hepatocellular carcinoma
Group Members
Group Members
Neeta Khandekar (Senior Research Assistant)
Cristian David Pena Martinez (Post-Doctoral Scientist)
Bradley Hall (PhD student)
Ann-Maree Huang (PhD Student)
Imogen Astruc (PhD Student)
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