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Development of hybrid AAV/HSV vectors for gene delivery to skeletal muscle: applications in the treatment of MPS disease

Reference	GTH12543
Principal Investigator / Supervisor	Dr R Coffin
Co-Investigators / Co-Supervisors	Professor Adrian Thrasher, Professor Bryan Winchester
Institution	University College London
Department	Immunology and Molecular Pathology
Funding type	Research
Value (£)	172,736
Status	Completed
Type	Research Grant
Start date	01/04/2000
End date	01/04/2003
Duration	36 months

Abstract

The study aims to provide improved technology for the production of adeno- associated virus (AAV) vectors using herpes simplex virus (HSV) rather than the more usual adenovirus for virus growth. It is aimed to develop a transfection-free AAV production method using hybrid AAV/HSV constructs and viruses which, unlike current systems, will allow effective scale-up of AAV production. AAV is highly promising for gene delivery to muscle. The project aims to test AAV vectors for treatment of Hurler's disease (an MPS disease) in a knockout mouse model following gene delivery to skeletal muscle. Such an approach is promising as the deficient enzyme in Hurler's disease is naturally secreted from expressing cells, and moreover can be re-absorbed elsewhere to hopefully provide a therapeutic effect.

Summary

unavailable

Committee	Closed Committee - Genes & Developmental Biology (GDB)
Research Topics	X – not assigned to a current Research Topic
Research Priority	X – Research Priority information not available
Research Initiative	Gene Technologies Underpinning Healthcare (GTH) [1999]
Funding Scheme	X – not Funded via a specific Funding Scheme

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