A Helper-Dependent Adenoviral Gene Therapy Delivery and Expression System

a technology of adenoviral gene therapy and adenoviral gene, applied in the direction of dsdna viruses, viruses/bacteriophages, genetic material ingredients, etc., can solve the problems of no curative treatment currently available, the long-term biological effect of prg4 over-expression and the molecular mechanism of its potential therapeutic benefits are still poorly understood, and the development of inhibitors of such pathways has not proven effective in clinical settings. to achieve the effect of prevention and/or
US20150361452A1Inactive Publication Date: 2015-12-17BAYLOR COLLEGE OF MEDICINE
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Patent Information

Authority / Receiving Office
US · United States
Current Assignee / Owner
BAYLOR COLLEGE OF MEDICINE
Publication Date
2015-12-17
Estimated Expiration
Not applicable · inactive patent

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Abstract

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.
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Description

FIELD OF THE INVENTION

[0001] 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.DESCRIPTION OF THE BACKGROUND ART

[0002] Musculoskeletal conditions are the most common chronic conditi...

Claims

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