Gene therapy can be used in the treatment of many diseases - cancer, AIDS or cardiovascular disorders. Its limited clinical applications mainly due to the difficulties associated with the introduction of genes into tissues and obtaining them effective expression. To date, most gene therapy protocols using a viral system. More often considered the possibility of using a vector constructed from the adeno-associated virus (adeno-associated virus, AAV). AAV vectors to efficiently infect cells that are dividing and non-dividing, integrate into the host genome.

Over the last decade was observed significant development of gene therapy strategies in the hematopoietic system from attempts to correct the mutated gene in monogenic diseases to research on the use of gene transfer in the transplant , the treatment of cancer and AIDS. Intensive research therapy based on genetically modified stem cells - the results obtained in animal models to date are encouraging. The use of gene therapy requires improvements in gene transfer methods and to ensure their safety.