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The ability to conduct mechanistic experiments is essential for understanding the functions of disease-associated genes. However, many in vivo approaches for gene manipulation remain prohibitively expensive. Adeno-Associated Viruses (AAVs) offer a cost-effective and time-efficient alternative, enabling targeted gene delivery to specific tissues and cell types. This powerful tool has become increasingly important for researchers aiming to investigate gene function with precision and scalability.

Supported by the Jennie Mackenzie Research Fund, the Charles Perkins Centre AAV Accelerator provides researchers with expert guidance and practical support in the use of AAVs, and position the Charles Perkins Centre as a leader within the field of gene therapy.

The CPC AAV Accelerator

• improves the reproducibility of AAV-based experiments by establishing standardised operating procedures
• provides hands-on expertise to assist researchers to design tissue-specific expression vectors
• subsidises AAV manufacturing costs for scientists at the Charles Perkins Centre
• pioneers innovative experimental designs using AAVs. 

We are actively involved in developing new methods to study complex metabolic diseases. Although AAVs are most commonly used to increase gene expression, the discovery of miniature CRISPR enzymes now means that AAVs can also be used to ablate gene expression. This is critical to enable researchers to fully investigate the functions of disease associated genes. To enable researchers to better understand how specific tissues contribute to disease, we are developing high performance promoter elements to target AAVs to specific cell types.

Establishing these capabilities is essential to overcoming one of modern medicine’s greatest challenges – understanding why certain individuals do not respond to otherwise effective front-line therapeutics. It is commonly accepted that human disease results from a dynamic interplay between genetic and lifestyle factors. However, the limitations of established methods for studying the functions of disease associated genes have meant that almost all preclinical experiments and gene function studies have been conducted in single (genetically identical) strains of mice. Consequently, research outcomes rarely reflect the full complexity of human biology. We are committed to overcoming this limitation through the use of AAVs.

We are committed to advancing the Charles Perkins Centre’s mission to understand and combat chronic disease such as diabetes, obesity and cardiovascular disease. Developing improved therapies for these conditions will require deep understanding of the biological mechanisms that underpin them. AAV-based approaches offer an affordable and efficient means by which to conduct high-impact experiments and discover new avenues to progress therapeutic development. 

To streamline the manufacturing of AAVs, we have partnered with the  at the Children’s Medical Research Institute. This collaborative arrangement leverages the respective expertise of the Vectorology Facility and the Charles Perkins Centre to maximise our research impact.

Whether you’re in the process of conducting AAV-based experiments already, or just want to find out where to begin, we’re here to help.

Here’s how it works:

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• as soon as we can, we’ll respond with any resources that address your questions
• if you need hands-on help, we’ll organise a 30-minute consultation to discuss your specific project requirements and discuss how we can best collaborate to achieve your research goals. 

• Dr Harry Cutler | Director
• ¶Ù°ùÌý | Vectorolgy Facility Lead, Children’s Medical Research Institute
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