The gene delivery system for rheumatoid synovium

Area of Science:

• * Gene therapy for rheumatoid arthritis (RA).
• * Molecular biology and virology.
• * Translational research in rheumatology.

Background:

• * Rheumatoid arthritis (RA) is a chronic inflammatory disease affecting synovial joints.
• * Gene therapy aims to induce expression of anti-arthritic molecules in target cells.
• * Viral vectors, such as adenovirus, have shown effectiveness in synovial tissue gene transfer.

Purpose of the Study:

• * To review properties and effectiveness of gene transfer methods for synovial tissue in RA.
• * To discuss investigations into gene expression in synovial tissue and cells.
• * To evaluate the potential of gene therapy for RA treatment.

Main Methods:

• * Classification of gene transfer methods into virus vectors and virus-free vectors.
• * Review of previous studies on viral vector-mediated gene transfer to synovial tissue.
• * Discussion of adeno-associated virus (AAV) mediated gene therapy and novel virus-free vectors.

Main Results:

• * Viral vectors, particularly adenovirus-based, are effective for gene transfer in synovial tissue.
• * Virus vectors are considered more applicable for clinical approaches, with AAV trials initiated in 2007.
• * Numerous effective novel virus-free vectors have been developed.

Conclusions:

• * Gene therapy is a promising, potentially novel therapeutic strategy for rheumatoid arthritis.
• * Current gene transfer technologies require further safety improvements for clinical application.
• * Gene therapy serves as an ideal technique for "Bench to Clinic and Clinic to Bench" research in RA.