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

Inactive Publication Date: 2015-12-17
BAYLOR COLLEGE OF MEDICINE
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Benefits of technology

[0012]It is therefore an object of the present invention to provide an improved delivery and expression system that allows for long-term expression of biologically active proteoglycan 4 (PRG4) for use in the prevention an...

Problems solved by technology

Unfortunately, despite significant investment, the development of inhibitors of such pathways has not proven effective in the clinical setting.
However, the long-term biological effects of PRG4 over-expression and the molecular mechanism of its potential therapeutic benefits are still poorly understood.
Although some approaches suggest gene therapy for treating or preventing joint disorders such as osteoarthritis, no curative treatments are currently available.
However, while these drugs can suppress joint inflammation, many of them are known to have deteriorating effects on the cartilage, which further worsens the underlying process of osteoarthritis development.
Furthermore, polysulphated glycosaminoglycans injected into the joint or intramuscularly as well as orally admini...

Method used

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  • A Helper-Dependent Adenoviral Gene Therapy Delivery and Expression System
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  • A Helper-Dependent Adenoviral Gene Therapy Delivery and Expression System

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Results

PRG4 Prevents Development of Age Related Osteoarthritis Changes

[0040]To investigate the long-term effect of Prg4 over-expression, the inventors generated transgenic mice expressing Prg4 under the cartilage specific type II collagen promoter (Col2a1) (FIG. 5A). PRG4 transgenic mice expressed Prg4 ectopically in growth plate cartilage and over-express Prg4 in articular cartilage throughout development and adulthood (FIG. 5B-D). Macroscopically, the inventors detected no differences in growth or skeletal development in Prg4 transgenic mice when assessed by weight (FIG. 5E). Microscopically, markers of chondrocyte proliferation or apoptosis remained the same in Prg4 mice vs. wild type mice as assessed by BrdU staining (p=n.s. in both proliferating and resting zone of P1 chondrocytes) (FIG. 5F) and TUNEL staining (no positive signals in P1 chondrocytes) (FIG. 5G), respectively. These data suggested that ectopic over-expression of Prg4 in cartilage did not significantly affect chon...

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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.

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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Application Information

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IPC IPC(8): C12N15/86
CPCC12N15/86A61K48/0058C12N2710/10043C12N2710/10343C12N2800/24
Inventor RUAN, ZHECHAOGUSE, KILIANLEE, BRENDAN
Owner BAYLOR COLLEGE OF MEDICINE
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