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Musculoskeletal disease

Osteoarthritis (OA)

Proteoglycan 4 (PRG4; HAPO)

A study in mice suggests enhancing levels of PRG4 in joints could help treat OA. In aging mice, Prg4 overexpression in cartilage tissue decreased age-related OA compared with normal Prg4 expression. In mice subjected to knee ligament transection, local delivery of an adenoviral vector containing Prg4 decreased OA-associated joint pathology compared with delivery of a sham vector. Next steps include testing the Prg4 gene therapy in a horse model of OA.
GeneQuine Biotherapeutics GmbH's GQ-203 is a gene therapy using the same vector as in the study but containing a different, undisclosed gene. It is in preclinical development to treat OA.

SciBX 6(12); doi:10.1038/scibx.2013.293
Published online March 28, 2013

Findings covered by provisional patents; licensed to GeneQuine Biotherapeutics

Ruan, M.Z.C. et al. Sci. Transl. Med.; published online March 13, 2013;
Contact: Brendan H.L. Lee, Baylor College of Medicine, Houston, Texas