Gene therapy involves the transfer of genetic material into a patient’s cells in order to correct a disease state. The objective is to provide the necessary genetic material to correct a genetic deficiency and allow a particular therapeutic protein to be temporarily or permanently expressed. Candidates for gene therapy are often missing a particular gene or have deficiencies in their genome preventing them from producing fully functional, therapeutic proteins. Examples can include hemophiliacs (missing blood clotting factors) and diabetics (missing or producing insufficient quantities of insulin). With the increased understanding of the genetic basis of disease and the Human Genome Project, the potential for new gene therapy treatments is rapidly increasing.
As referred to above, gene therapy requires inserting genetic material into a patient’s genome. Beyond obtaining the proper sequence of DNA, the insertion process requires a mechanism for allowing the donor DNA to enter the patient’s target cells. There are a number of vehicles for transferring DNA, the most common methods being Adenovirus, Adeno-Associated Virus, synthetic vectors (liposomes), and DNA directly.
This 3M Purification Application Brief addresses filtration applications employed in the production and purification of Adenoviral vectors used for gene therapy processes.