Chapter 8 - Gene Therapy and Pharmacogenomics

Acquired disease

Any disease triggered by external factors and not directly caused by a person’s genes. (e.g., an infectious disease, noncongenital cardiovascular diseases)

Alleles

The two or more alternative forms of a gene.

Chromosomes

Structures in the nuclei of cells that contain threads of DNA, which transmit genetic information.

Gene

The biologic unit of heredity; a segment of a DNA molecule that contains all of the molecular information required for the synthesis of a biologic product such as proteins or RNA molecules or an amino acid chain (protein molecule)

Gene therapy

New therapeutic technologies that directly target human genes in the treatment or prevention of illness.

Genetic disease

Any disorder caused directly by a genetic mechanism.

Genetic material

DNA or RNA molecules or portions thereof.

Genetic polymorphisms (PMs)

Variants that occur in the chromosomes of 1% or more of the general population.

Genetic predisposition

Factors in a person’s genetic makeup that increases their likelihood of developing one or more diseases.

Genetics

The study of the structure, function, and inheritance of genes.

Genome

The complete set of genetic material of any organism.

Genomics

The study of the structure and function of the genome, and the way genes and their products work.

Genotype

The particular alleles present at a given site on the chromosomes that determine a specific genetic trait.

Heredity

The characteristics and qualities that are genetically passed from one generation to the next.

Human Genome Project (HGP)

A scientific project to describe in detail the entire genome of a human being.

Inherited disease

Genetic disease that results from defective alleles passed from parents to offspring.

Nucleic acids

Molecules of DNA and RNA in the nucleus of every cell.

Personalized medicine

The use of molecular and genetic characterizations of the disease process and the patient for custom drug therapy.

Pharmacogenetics

The study of the genetic basis for variations in the body’s response to drugs.

Pharmacogenomics

A branch of pharmacogenetics that surveys the entire genome to detect multigenic determinants of drug response.

Phenotype

The expression in the body of a genetic trait that results from a person’s particular genotype.

Recombinant DNA (rDNA)

DNA molecules that have been artificially synthesized or modified in a laboratory setting.