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Gene therapy for osteoarthritis


Gene transfer strategies for medical management of the Osteoarthritis have attracted the attention of scientists due to the complex pathology of this chronic disease. Unlike other pharmacological treatments, gene therapy targets the disease process rather than the symptoms.

Passing from parents to children, genes are the building blocks of inheritance. They contain instructions for making proteins. If genes do not produce the right proteins in a correct way, a child can have a genetic disorder. Gene therapy is a molecular method aiming to replace defective or absent genes, or to counteract the ones undergoing overexpression. For this purpose, three techniques may be utilized: gene isolation, manipulations, and transferring to target cells. The most common form of gene therapy involves inserting a normal gene to replace an abnormal gene. Other approaches including repairing an abnormal gene and altering the degree to which a gene is turned on or off. Two basic methodologies are utilized to transfer vectors into target tissues; Ex vivo gene transfer and In-vivo gene transfer. One type of gene therapy in which the gene transfer takes place outside the patient's body is called ex vivo gene therapy. This method of gene therapy is more complicated but safer since it is possible to culture, test, and control the modified cells.

Osteoarthritis (OA) is a degenerative joint disease which is the western world's leading cause of pain and disability. It is characterized by the progressive loss of normal structure and function of articular cartilage, the smooth tissue covering the end of the moving bones. This chronic disease not only affects the articular cartilage but the subchondral bone, the synovium and periarticular tissues are other candidates. People with OA can experience severe pain and limited motion. OA is mostly the result of natural aging of the joint due to biochemical changes in the cartilage extracellular matrix.

Osteoarthritis is caused by mechanical factors such as obesity, joint trauma, mechanical overloading of joints or joint-instability.Genetics is also a leading factor that contributes to OA. Studies have shown that genetics is the source of at least 50% of OA cases in the hands and hips. Since the degeneration of cartilage is an irreversible phenomenon, it is incurable, costly and responds poorly to treatment. Due to the prevalence of this disease nowadays, the repair and regeneration of articular cartilage has become a dominant area of research. The growing number of the patients suffering from osteoarthritis and the effectiveness of the current treatments attract a great deal of attention to genetic-based therapeutic methods to cure and prevent the progression of this chronic disease.


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