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Development of Tissue-Targeted Vectors for Musculoskeletal Gene Therapy


A unique opportunity is available for an outstanding PhD scholar to conduct research in developing tissue-targeted AAV vectors for gene delivery and CRISPR gene editing.


Associate Professor Aaron Schindeler.

Research location

Westmead - Childrens Hospital at Westmead Clinical School

Program type



Discovering new and improved therapies for musculoskeletal (MSK) conditions in children can have a tremendous health impact on their lifetime wellbeing and productivity. MSK health is a key NHMRC national priority area. There are over 160 genetic bone disorders with the most common being Osteogenesis Imperfecta (OI; brittle bone disease), which affects 1:20,000 live births. Gene therapy offers the first real promise for a curative therapy for these genetic diseases and our team has already shown proof-of-concept for new technological advances that can transform the field. The aim of this PhD Scholarship project will be to develop new gene therapy approaches for the therapeutic rescue of genetic musculoskeletal diseases, using targeted high-efficiency AAV vector systems.
The successful candidate will have a strong understanding of the principles of genetics and molecular biology along with a complementary research track record. Prior experience working with mouse models or adeno-associated virus vectors will be strongly favoured. Applicants are expected to have excellent written and spoken communication skills.

Additional information

Scholarship Opportunity: This position is supported by an NSW HEALTH GENE & CELL THERAPY PHD PROGRAM SCHOLARSHIP funded by NSW Health and the University of Sydney. It provides for a stipend for $40,000 p.a. for the duration of the student's higher degree.

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Opportunity ID

The opportunity ID for this research opportunity is 2927

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