The potential for therapy of immune disorders with gene therapy

Gene therapy often is considered the ultimate form of therapy, based on its potential for correcting genetic diseases at the molecular level as opposed to merely treating the symptoms of the defect. Primary disorders of immunity have played a major role in the development of gene therapy, with the first human experiment being performed on ADA-deficient patients in 1990 and the first clear clinical benefit of genetic correction being reported in X-Linked severe combined immunodeficiency patients in 2000. After a decade of clinical experimentation, the challenges and potential of gene therapy for immunodeficiencies are better defined and are discussed in this article together with a brief description of the techniques that commonly are used for corrective gene transfer.