Technologies

Current State of the Art

Gene therapy involves the use of introducing healthy genes into a patient whose own genes are defective. In theory, the genes introduced will combine with the patient’s DNA in targeted cells to trigger production of healthy cells. Gene therapy has become a viable medical treatment option for certain diseases. Many current gene therapies use foreign DNA or viral vectors. Applications exist for oncology, cystic fibrosis, Parkinson’s disease, Huntingon’s disease, retinal degeneration, pancreatitis, AIDS and sensory disorders such as loss of sight and sound.

Problems with the Current Art

Although some cellular gene therapy products have been approved, only limited human gene therapy products have yet been approved, none by the USFDA and only some in Europe and China. The primary reason for this is that the foreign DNA or viral vectors introduced by the gene therapy create competing pathway uptake, negative immune responses or undesirable gene mutations. Some of the problems with gene therapies include the limited length of time the gene expresses itself, having the viral vector regain its virulence once introduced into a host organism, or finding the most effective method of delivering the gene to target cells.