theoretically modify specific genes resulting in disease cure following
a single administration. Initially gene therapy was envisioned for the
arthritis, neurodegenerative disorders and other acquired diseases.
relevant gene must be identified and cloned. Upon completion of the
Human Genome Project, gene availability will be unlimited, but until
identification and cloning for relevant genes related to the disease.
Once the gene has been identified and cloned, the next consideration
transfer and gene expression remain at the forefront of gene therapy
obtaining sufficient levels of expression for disease treatment.
expression will resolve these issues in the majority of gene therapy
protocols. Other important considerations for a gene therapy strategy
disorder, potential side effects of the gene therapy treatment, and
understanding of the target cells to receive the gene therapy.
Gene transfer vector is the mechanism by which the gene is transferred
protocols worldwide. Since the approval process for these protocols is
not as public outside the U.S., it is difficult to obtain an exact
number of worldwide protocols. Of the publicized protocols, 125 are
China and Japan. As of 31 December 1995, 1024 patients had been treated
in either a gene transfer or gene therapy protocol. Much controversy
exists regarding how many of these have benefited from their gene
therapy, and no one has yet been cured.
Public controversy in the field of human gene therapy is driven by
understand the enormous potential of gene therapy, but the former may
not appreciate all the pitfalls and uncertainly that lies in the
immediate future. The financial interests of biotechnology firms and,
some have asserted, the career interests of some gene therapists have
encouraged extravagant, or at least overly optimistic, public
statements about contemporary gene therapy. In spite of the
proliferation of protocols, the actual number of patients treated
remains small, and only one genuinely controlled study of human gene
therapy has been published as of this date.
Anderson WF. Human Gene Therapy. 1995; 6:1505-1506.
Report and Recommendations of the Panel to Assess the NIH Investment in
Research on Gene Therapy Available at: