Loading patent…
AssigneeBaylor College of Medicine
InventorsZhechao Ruan, Kilian Guse, Brendan Lee
Estimating the term…
The present invention relates to gene therapy delivery and expression systems comprising at least one helper-dependent adenoviral vector containing a nucleic acid sequence encoding for proteoglycan 4 (PRG4) or a biologically active fragment thereof. The invention further relates to a pharmaceutical composition comprising a therapeutically effective amount of at least one helper-dependent adenoviral vector containing said nucleic acid sequence encoding for proteoglycan 4 (PRG4), or a homolog thereof from any other species, or a biologically active fragment thereof. The invention also relates to the use of the novel gene therapy delivery and expression system according to the invention for use in the prevention and/or treatment of camptodactyly-arthropathy-coxa vara-pericarditis (CACP), or a musculoskeletal disorder such as a joint disorder or joint disease.
A method for the treatment of osteoarthritis in a subject in need thereof, comprising administering to a joint of the subject a helper-dependent adenoviral vector comprising a nucleic acid sequence encoding proteoglycan 4 (PRG4), a left inverted terminal repeat (LITR) and a right adenoviral inverted terminal repeat (RITR), adenoviral packaging signal sequences and non-viral, non-coding stuffer nucleic acid sequences, wherein PRG4 expression is controlled by an elongation factor 1 alpha (EF 1 alpha) promoter,
A method for the prevention of post-traumatic osteoarthritis in a subject in need thereof, comprising administering to a joint of the subject a helper-dependent adenoviral vector comprising a nucleic acid sequence encoding PRG4, a LITR and a RITR, adenoviral packaging signal sequences and non-viral, non-coding stuffer nucleic acid sequences, wherein PRG4 expression is controlled by an elongation factor 1 alpha (EF 1 alpha) promoter, and
Dependencies are inferred from the claim text.