A5040764876- Virus-based gene therapy research
- Amyotrophic Lateral Sclerosis Research
- Neurogenetic and Muscular Disorders Research
- Genetic Neurodegenerative Diseases
- Viral gastroenteritis research and epidemiology
- Viral Infections and Immunology Research
- RNA Interference and Gene Delivery
- Herpesvirus Infections and Treatments
- Bacterial Genetics and Biotechnology
- Bacterial biofilms and quorum sensing
- Biochemical and Structural Characterization
- CRISPR and Genetic Engineering
- Organ and Tissue Transplantation Research
- RNA Research and Splicing
- Dermatology and Skin Diseases
- Poxvirus research and outbreaks
- Nerve injury and regeneration
- Analytical Chemistry and Sensors
- Animal Virus Infections Studies
The University of Sydney
2023-2024
Children's Medical Research Institute
2023-2024
University of Pittsburgh
2024
Amyotrophic Lateral Sclerosis Association
2024
Westmead Institute for Medical Research
2023
University of Technology Sydney
2019-2020
Abstract Developing clinically predictive model systems for evaluating gene transfer and editing technologies has become increasingly important in the era of personalized medicine. Liver-directed therapies present a unique challenge due to complexity human liver. In this work, we describe application whole liver explants an ex situ normothermic perfusion system evaluate set fourteen natural bioengineered adeno-associated viral (AAV) vectors directly liver, presence absence neutralizing sera....
Directed evolution of natural AAV9 using peptide display libraries have been widely used in the search for an optimal recombinant AAV (rAAV) transgene delivery across blood-brain barrier (BBB) to CNS following intravenous ( IV) injection. In this study, we a different approach by creating shuffled rAAV capsid library based on parental serotypes 1 through 12. Following selection mice, 3 novel variants closely related AAV1, AAV-BBB6, AAV-BBB28, and AAV-BBB31, emerged as top candidates. direct...
Gene therapies and associated technologies are transforming biomedical research enabling novel therapeutic options for patients living with debilitating incurable genetic disorders. The vector system based on recombinant adeno-associated viral vectors (AAVs) has shown great promise in recent clinical trials diseases of multiple organs, such as the liver nervous system. Despite successes toward development bioengineered AAV variants improved transduction primary human tissues cells, that can...
TDP-43 mislocalization, aggregation, and loss of splicing function are neuropathological hallmarks in over 97% Amyotrophic Lateral Sclerosis (ALS), 45% Frontotemporal Lobar Degeneration (FTLD), 60% Alzheimer's Disease, which has been reclassified as LATE-NC. However, the mechanisms underlying dysfunction remain elusive. Here, we utilize APEX2-driven proximity labeling mass spectrometry to characterize context-dependent interactome conditions cytoplasmic impaired RNA-binding contributing...
Viral vectors based on recombinant adeno-associated virus (rAAV) have become the most widely used system for therapeutic gene delivery in central nervous (CNS). Despite clinical safety and efficacy neurological applications, a barrier to adoption of current generation lies their limited efficiency, resulting transduction CNS target cells. To address this limitation, researchers bioengineered fit-for-purpose AAVs with improved tropism tissue penetration. While preclinical assessment these...
Herpes simplex virus-1 (HSV-1) is a human pathogen known to cause cold sores and genital herpes. HSV-1 establishes lifelong infections in our sensory neurons, with no cure or vaccine available. can reactivate sporadically travel back along nerves, where it form lesions the oral mucosa, eye, skin, be shed asymptomatically. New treatment options are needed as resistance emerging current antiviral therapies. Here, we show that interferons (IFNs) capable of blocking virus release from nerve...
Twitching motility-mediated biofilm expansion occurs via coordinated, multi-cellular collective behaviour to allow bacteria actively expand across surfaces. Type-IV pili (T4P) are cell-associated virulence factors which mediate twitching motility rounds of extension, surface attachment and retraction. The Chp chemosensory system is thought respond environmental signals regulate the biogenesis, assembly function T4P. In other well characterised systems, methyl-accepting chemotaxis proteins...
Given the mounting evidence implicating TDP-43 dysfunction in several neurodegenerative diseases, there is a pressing need to establish accessible tools sense and quantify loss-of-function (LOF). These are crucial for assessing potential disease contributors exploring therapeutic candidates proteinopathies. Here, we develop sensitive accurate real-time sensor LOF: CUTS (CFTR UNC13A Loss-of-Function) system. This system combines previously reported cryptic exons regulated by with reporter,...
Given the mounting evidence implicating TDP-43 dysfunction in several neurodegenerative diseases, there is a pressing need to establish accessible tools sense and quantify loss-of-function (LOF). These are crucial for assessing potential disease contributors exploring therapeutic candidates proteinopathies. Here, we develop sensitive accurate real-time sensor LOF: CUTS (CFTR UNC13A Loss-of-Function) system. This system combines previously reported cryptic exons regulated by with reporter,...
Given the mounting evidence implicating TDP-43 dysfunction in several neurodegenerative diseases, there is a pressing need to establish accessible tools sense and quantify loss-of-function (LOF). These are crucial for assessing potential disease contributors exploring therapeutic candidates proteinopathies. Here, we develop sensitive accurate real-time sensor LOF: CUTS (CFTR UNC13A Loss-of-Function) system. This system combines previously reported cryptic exons regulated by with reporter,...
Abstract Developing clinically predictive model systems for evaluating gene transfer and editing technologies has become increasingly important in the era of personalized medicine. Liver-directed therapies present a unique challenge due to complexity human liver. In this work, we describe application whole liver explants an ex situ normothermic perfusion system evaluate set fourteen natural bioengineered adeno-associated viral (AAV) vectors directly liver, presence absence neutralizing sera....
Abstract Developing clinically predictive model systems for evaluating gene transfer and editing technologies has become increasingly important in the era of personalized medicine. Liver-directed therapies present a unique challenge due to complexity human liver. In this work, we describe application whole liver explants an ex situ normothermic perfusion system evaluate set fourteen natural bioengineered adeno-associated viral (AAV) vectors directly liver, presence absence neutralizing sera....
Abstract Twitching motility-mediated biofilm expansion occurs via coordinated, multi-cellular collective behaviour to allow bacteria actively expand across surfaces. Type-IV pili (T4P) are cell-associated virulence factors which mediate this rounds of extension, surface attachment and retraction. The Chp chemosensory system is thought respond environmental signals regulate the biogenesis, assembly twitching motility function T4P. In other well characterised systems, methyl-accepting...