Gene therapy for skeletal disease in MPS.

Funded Activity Summary

Skeletal disease is a major problem for children with mucopolysaccharidoses (MPS). Patients suffer from early onset osteoporosis and osteoarthritis, severely affecting their quality of life. We will evaluate a lentiviral gene therapy vector developed in-house for its capacity to transduce bone, cartilage, synovial and ligament cells in a mouse model of MPS VI. Our goal is to generate high level, sustained expression of the deficient MPS enzyme and alter the course of skeletal disease in MPS.

Funded Activity Details

Start Date: 01-01-2009

End Date: 01-01-2011

Funding Scheme: NHMRC Project Grants

Funding Amount: $383,783.00

Funder: National Health and Medical Research Council

Research Topics

ANZSRC Field of Research (FoR)

Paediatrics

ANZSRC Socio-Economic Objective (SEO)

There are no SEO codes available for this funding activity

Other Keywords

degenerative joint disease | gene therapy | inherited metabolic disease | lysosomal storage disorder | mucopolysaccharidoses | musculoskeletal disease | orthopaedics | skeletal dysplasia

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