Building Better People: The Truths and Myths of Gene Therapy

Article excerpt

Biotechnology represents one of the most technically challenging issues for journalists to cover in the news. Not only is the subject complex, but rapid changes and advances in the various biotech fields provide an additional challenge for journalists. The Foundation for American Communications recently held a conference at the Harrison Conference Center near Chicago to assist reporters in their daunting assignment. FACS brought together a faculty of top researchers in the field, university professors, clinical researchers, medical ethicists and industry experts. The group covered subjects ranging from stem cell research, gene therapy and cloning to the related law and ethics. Provided here is coverage of the discussion of gene therapy, its history and regulations.

The sound of terms like "gene therapy" and "recombinant DNA technology" might conjure up negative images of eugenics, clandestine government experiments and conspiracies. For that reason, Dr. Nelson Wivel, deputy director of the Institute of Gene Therapy at the University of Pennsylvania School of Medicine, favors a public oversight system that allows free access to the media and the rest of the general public. Wivel, former director of the National Institutes of Health's Recombinant DNA Advisory Committee (RAC), is an expert on the history of gene therapy and its regulation. Wivel spoke at a recent FACS biotech conference.

"As long as the public has access to open discussion of new issues in gene therapy, and as long as the press reports those discussions, there is reason to be optimistic that the science will eventually go forward," Wivel said.

Researchers have been contemplating gene therapy for the past 50 years. The first clinical gene therapy trial took place in 1990 with the study of a patient with severe combined immune deficiency (SCID).

"The patient was not cured, but proof of principle was established in that successful gene transfer was demonstrated," Wivel said.

The first evidence of successful gene therapy was reported in 2000 by French researcher Alain Fisher in the treatment of SCID. The DNA research community was rocked in 1999 when Jesse Gelsinger died during gene therapy administered at the University of Pennsylvania School of Medicine.

Since then, researchers and regulators have regrouped and reorganized, and research is proceeding again on a more cautious course.


Wivel described gene therapy as the insertion of functioning genes into a person's cells, either to correct an inborn genetic error or to impart a new function to the cell. Somatic cell gene therapy, as it is called, is an application of recombinant DNA technology. Somatic cells are non-reproductive cells, as opposed to germ-line cells which are reproductive cells.

Recombinant DNA refers to DNA molecules in which sequences which are not naturally contiguous have been placed next to each other by in vitro manipulations. The different sequences within a recombinant DNA molecule will frequently have come from entirely different organisms.

A critical component of gene therapy is the delivery of the genes to the body. Unlike typical drugs (Wivel considers gene therapy akin to drug therapy), genes cannot simply be taken like a pill. Genes have to be inserted into a cell's nucleus. During the 1960s, researchers contemplated the use of viruses, normally an enemy, as an agent for delivery of genes. Viruses replicate themselves by invading a cell's DNA and inserting their own genetic code.

"Viruses are the smartest cell parasites that we know about: They're clever; they know how to replicate; and they can be re-engineered to make them non-infectious," Wivel said.

In gene therapy, the desired genes must first be isolated from a normal chromosome; and then those genes are attached to the DNA of a harmless virus, which is then let loose on the patient. …