Building Better People: The Truths and Myths of Gene Therapy

By Murray, Bruce | The Quill, June 1, 2001 | Go to article overview

Building Better People: The Truths and Myths of Gene Therapy


Murray, Bruce, The Quill


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.

WHAT IS GENE THERAPY AND HOW IS IT DONE?

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.

Animal gene therapy trials are currently underway as possible therapy for cystic fibrosis. "The testing of adenovirus vectors in primates and mice is a necessary preclinical step, but it has shortcomings in that these vertebrates are not natural hosts for this particular virus," Wivel said.

ETHICAL ISSUES

In 1980, a consortium of Protestant, Catholic and Jewish groups sent a letter to President Jimmy Carter expressing concerns about genetic engineering and maintaining the fundamental nature of human life and dignity of the individual.

Safety and ethical debates among scientists preceded that, culminating in 1976 with the release of the "NIH Guidelines for Research Involving Recombinant DNA Molecules," also known at "the phone book" for its exhaustive length.

Debate leading up to creation of the document was sparked by a breakthrough in developing a viral vector. …

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