Gene therapy for skeletal disease in MPS. [ 2009 - 2011 ]

Also known as: Gene therapy for bone and cartilage disease in MPS.

Research Grant

[Cite as]

Researchers: Dr Sharon Byers (Principal investigator) ,  A/Pr Donald Anson

Brief description 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.

Funding Amount $AUD 383,783.50

Funding Scheme NHMRC Project Grants

Notes Standard Project Grant

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