Promise of Gene Therapy

There was extensive media coverage and public excitement about the promise of gene therapy as the first clinical trials commenced in 1990. In fact, many individuals and families with genetic disorders expected an imminent cure for their diseases. Unfortunately, there are as yet few successes to report, even though hundreds of somatic cell gene therapy clinical trials for many different diseases have been attempted. The early hype of gene therapy might have been avoided with more open and honest communication about gene therapy and its expectations between the researchers, physicians, patients, and the public. The death of the participant in 1999 and deficiencies in the protocol (study design) used for that trial underscore the need for continued public discourse on gene therapy.

The promise of gene therapy has not diminished, even though its full therapeutic potential is not yet known. Scientists, physicians, patients, and families continue to look forward to many future successes for gene therapy. With the completion of the Human Genome Project and the accelerated discovery of human disease genes, the potential number of diseases for which gene therapy could be beneficial continues to increase. With further research into the technical aspects of gene therapy and continued public debate about the ethical issues involved in such treatments, it is hoped gene therapies will become standard, effective treatments in the next few decades. see also Cystic Fibrosis; Eugenics; Gene Therapy; Growth Disorders; Muscular Dystrophy; Prenatal Diagnosis; Severe Combined Immune Deficiency.

Elizabeth C. Melvin


Anderson, W. French. "Human Gene Therapy." Nature 392 (1998): 25-30.

Smaglik, Paul. "Congress Gets Tough with Gene Therapy." Nature 403 (2000): 583-584.

Walters, Leroy. "The Ethics of Human Gene Therapy." Nature 320 (1986): 225-227.

Internet Resources

American Society of Human Gene Therapy. <>.

"Gene Therapy." Human Genome Program of the U.S. Department of Energy. <>.

Genetic Code

The sequence of nucleotides in DNA determines the sequence of amino acids found in all proteins. Since there are only four nucleotide "letters" in the DNA alphabet (A, C, G, T, which stand for adenine, cytosine, guanine, and thymine), but there are 20 different amino acids in the protein alphabet, it is clear that more than one nucleotide must be used to specify an amino acid. Even two nucleotides read at a time would not give sufficient combinations (4 X 4 = 16) to encode all 20 amino acids plus start and stop signals. Therefore it would require a minimum of three DNA nucleotides nucleotides the build ing blocks of RNA or DNA


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