engleski

Vectors in Gene Therapy

The discovery of genetic cause of numerous diseases provided the molecular basis for gene therapy. The concept of gene therapy involves the introduction of the desired gene whose product should cure or slow down the progression of disease. This approach of curing requires the efficient gene transfer in a variety of target cells, tissues and organs. The delivery vehicles, called vectors, are the critical points of gene therapy, and still its “ Achilles heel” . Therapeutic genes can be introduced into the target cells via either viral or non-viral vectors. Although non-viral vectors, including naked DNA and liposomes, are relatively easy to make and safe, they are still not sufficiently efficient and lack the ability to target specific cell type (19). Viral vectors are more suitable for efficient gene delivery because viruses are naturally evolved to transfer their genes into the host cells. This property made them desirable for engineering viral vector systems for the delivery of therapeutic genes. Viral vectors have the ability to specifically target certain cell types and tissues and to infect a high proportion of cells with high capacity for carrying transgenes in their genetically modified genome. Many different viral systems have been developed to transfer the desired genes. They are mainly derived from murine and human DNA- and RNA-viruses. The most commonly used vectors are derived from retroviruses, adenoviruses, herpes simplex virus (HSV) and adeno-associated virus (AAV) because they are well characterised. Recently, many different virus vectors are developing, derived from vaccinia virus, human cytomegalovirus (CMV), Epstein-Barr virus (EBV), poxviruses and foamy virus (18).

gene therapy; vector; retrovirus; adenovirus

nije evidentirano