Gene therapy, the replacement or supplementation of defective genetic information by transfer of normal functional genes, has significant potential as a method for treating genetic disorders. Recent approaches for gene therapy have involved gene transfer using recombinant viral vectors, exploiting RNA and DNA tumor viruses - many of which are pathogenic and capable of causing disease. Retroviruses have also been extensively studied, however, these viruses are unstable, randomly integrate into the host genome (which can lead to insertional mutagenesis) and may activate protooncogene expression. Adeno-associated viruses, also being utilized, have the advantage of integrating in specific regions of the host genome. The usefulness of currently available retroviral and adeno-associated viral vector systems is limited by their inability to accept heterologous DNA fragments greater than 3 to 5 kilobases (kb) and to produce large quantities of viral stocks. Recombinant adenovirus vector systems can now only accommodate 6 to 7 kb of foreign DNA and, despite deletion of certain viral replication regions, do cause significant cytopathology at high multiplicities of infection.
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