The challenge of gene therapy lies in development of a means to deliver the genetic material into the nuclei of the appropriate cells, so that it will be reproduced in the normal course of cell division and have a lasting effect. One technique involves removing cells from a patient, fortifying them with healthy copies of the defective gene, and reinjecting them into the patient. Another involves inserting a gene into an inactivated or nonvirulent virus and using the virus's infective capabilities to carry the desired gene into the patient's cells. A liposome, a tiny fat-encased pouch that can traverse cell membranes, is also sometimes used to transport a gene into a body cell. Another approach employing liposomes, called chimeraplasty, involves the insertion of manufactured nucleic acid molecules (chimeraplasts) instead of entire genes to correct disease-causing gene mutations. Once inserted, the gene may produce an essential chemical that the patient's body cannot, remove or render harmless a substance or gene causing disease, or expose certain cells, especially cancerous cells, to attack by conventional drugs.
Gene therapy was first used in humans in 1990 to treat a child with adenosine deaminase deficiency (ADA), a rare hereditary immune disorder (see immunity). It is hoped that gene therapy can be used to treat cancer, genetic diseases, and AIDS, but there are concerns that the immune system may attack cells treated by gene therapy, that the viral vectors could mutate and become virulent, or that altered genes might be passed to succeeding generations.
In the United States, gene therapy techniques must be approved by the federal government. The Recombinant DNA Advisory Committee of the National Institutes of Health oversees gene therapy experiments. Like drugs, products must pass the requirements of the Food and Drug Administration. Gene therapy is a competitive and potentially lucrative field, and patents have been awarded for certain techniques.
See J. Lyon and P. Gorner, Altered Fates: Gene Therapy and the Retooling of Human Life (1995).
Chemically, each gene consists of a specific sequence of DNA building blocks called nucleotides. Each nucleotide is composed of three subunits: a nitrogen-containing compound, a sugar, and phosphoric acid. Genes may vary in their precise makeup from person to person, including, for example, one nucleotide in a certain location in some people but another nucleotide in that location in others. Geometrically, the gene is a double helix formed by the nucleotides. Gene loci are often interspersed with segments of DNA that do not code for proteins; these segments are termed "junk DNA." When junk DNA occurs within a gene, the coding portions are called exons and the noncoding (junk) portions are called introns. Junk DNA makes up 97% of the DNA in the human genome, and, despite its name, is necessary for the proper functioning of the genes.
Each chromosome of each species has a definite number and arrangement of genes. Alteration of the number or arrangement of the genes can result in mutation. When the mutation occurs in the germ cells (egg or sperm), the change can be transmitted to the next generation. Mutations that affect somatic cells can result in certain cancers.
The scientific study of inheritance is genetics. The genetic makeup of an organism with reference to its set of genetic traits is called its genotype. The interaction of the environment and the genotype produces the observable attributes of the organism, or its phenotype. The sum total of the genes contained in an organism's full set of chromosomes is termed the genome. Scientists are working toward identifying the location and function of each gene in the human genome (see Human Genome Project). The decoding of the first free-living organism (a bacterium, Hemophilus influenzae) was completed in 1995 by J. Craig Venter and Hamilton Smith.
See also gene therapy; genetic engineering.
See biography by J. Cavanaugh (2006).
See biographies by C. Hirschhorn (1975) and A. Yudkoff (1999).
See his autobiography (1978); biography by H. George-Warren (2007); D. Rothel, The Gene Autry Book (1988).
Introduction of genetic material (by interbreeding) from one population of a species to another, thereby changing the composition of the gene pool of the receiving population. The introduction of new characteristics through gene flow increases variability within the population and makes possible new combinations of traits. In humans, gene flow usually comes about through human migration.
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Unit of heredity that occupies a fixed position on a chromosome. Genes achieve their effects by directing protein synthesis. They are composed of DNA, except in some viruses that contain RNA instead. The sequence of nitrogenous bases along a strand of DNA determines the genetic code. When the product of a particular gene is needed, the portion of the DNA molecule that contains that gene splits, and a complementary strand of RNA, called messenger RNA (mRNA), forms and then passes to ribosomes, where proteins are synthesized. A second type of RNA, transfer RNA (tRNA), matches up the mRNA with specific amino acids, which combine in series to form polypeptide chains, the building blocks of proteins. Experiments have shown that many of the genes within a cell are inactive much or even all of the time, but they can be switched on and off. Mutations occur when the number or order of bases in a gene is disrupted. Seealso genetic engineering, genetics, Hardy-Weinberg law, Human Genome Project, linkage group.
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Gene that determines whether or not a trait determined by another gene will be expressed. For example, when the gene responsible for albinism occurs, the genes that determine skin color are present but not expressed; the gene for albinism is therefore called an epistatic gene.
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(born May 25, 1898, New York, N.Y., U.S.—died Nov. 7, 1978, Greenwich, Conn.) U.S. boxer. Tunney boxed in the Marine Corps, earning the nickname “the Fighting Marine.” He defeated Jack Dempsey in 1926 to become the world heavyweight champion. In a controversial rematch in Chicago in 1927, Dempsey knocked Tunney to the canvas in the seventh round but failed to retire immediately to a neutral corner, thus delaying the count; the “long count” allowed Tunney to rise and win the 10-round fight. He retired the next year with a record of 65 wins in 77 bouts.
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(born Feb. 27, 1902, Harrison, N.Y., U.S.—died May 13, 1999, Naples, Fla.) U.S. golfer, prominent in the 1920s and '30s. Born to a poor Italian immigrant family, Sarazen began caddying when he was eight. He became a professional golfer in 1920. Sarazen was the first to win the four tournaments that comprise golf's grand slam in modern times. Sarazen won the U.S. Open (1922, 1932), the British Open (1932), and the PGA Championship (1922–23, 1933) and completed the grand slam with his Masters victory in 1935. That Masters victory was highlighted by his famous double eagle, two strokes (three under par) on the par-five 15th hole.
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(born Aug. 19, 1921, El Paso, Texas, U.S.—died Oct. 24, 1991, Santa Monica, Calif.) U.S. television and film producer. He worked as a pilot (1945–49) and police officer (1949–53) before becoming a writer for television series such as Dragnet and Dr. Kildare. He created the idea for the Star Trek series and produced the show from 1966 until it ended in 1969; later rerun in syndication, it developed a durable cult following among fans known as “Trekkies.” He produced six Star Trek movies, and from 1987 to 1991 he produced the television series Star Trek: The Next Generation.
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(born Aug. 23, 1912, Pittsburgh, Pa., U.S.—died Feb. 2, 1996, Beverly Hills, Calif.) U.S. dancer, choreographer, actor, and movie director. After training at his mother's dance school in Pittsburgh, he moved to New York in 1938 and danced in Broadway musicals, creating the h1 role in Pal Joey in 1940. Beginning in 1942, his athletic style and carefree acting—exemplified in the popular Anchors Aweigh (1945), On the Town (1949), An American in Paris (1951), and Singin' in the Rain (1952), which he also helped choreograph and direct—became hallmarks of the movie musical. His achievements earned him a special Academy Award in 1951. He later choreographed and directed numerous other movies and created a ballet for the Paris Opéra (1960).
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