Term Paper
GENE THERAPY
Gene therapy is based on the concept that genetic disorders and acquired diseases can be treated by replacing abnormal or absent genes or by modifying their functions. Inherited disorders such as cystic fibrosis and haemophilia, as well as catastrophic diseases such as cancer and AIDS, are prospective candidates for gene therapy. Although cures for these ailments would be welcome, some medical researchers suggest that the range of diseases that can be treated with gene therapy may be limited. In 2000, researchers used gene therapy techniques to help mice with haemophilia produce high levels of the protein needed to restore and maintain the clotting property of blood. For advocates, knocking out this disease in the human population makes gene therapy—despite its limitations— a worthwhile pursuit.
Gene therapy is the use of DNA as a pharmaceutical agent to treat disease. It derives its name from the idea that DNA can be used to supplement or alter genes within an individual's cells as a therapy to treat disease. The most common form of gene therapy involves using DNA that encodes a functional, therapeutic gene to replace a mutated gene. Other forms involve directly correcting a mutation, or using DNA that encodes a therapeutic protein drug (rather than a natural human gene) to provide treatment.
Gene therapy is composed of two categories: somatic gene therapy and germ line gene therapy. In somatic gene therapy, therapeutic genes are introduced to the diseased cells of a patient in hopes that they will genetically alter them to function normally. In germ line gene therapy, therapeutic genes are introduced to reproductive cells (egg and sperm cells) to prevent the manifestation of a genetic disorder before the patient is born. This approach would alter the patient’s genetic makeup and the genes he or she passes on to succeeding generations. Additionally, therapeutic genes can be introduced to cells in several ways. In ex vivo gene therapy, a patient’s
Gene therapy is based on the concept that genetic disorders and acquired diseases can be treated by replacing abnormal or absent genes or by modifying their functions. Inherited disorders such as cystic fibrosis and haemophilia, as well as catastrophic diseases such as cancer and AIDS, are prospective candidates for gene therapy. Although cures for these ailments would be welcome, some medical researchers suggest that the range of diseases that can be treated with gene therapy may be limited. In 2000, researchers used gene therapy techniques to help mice with haemophilia produce high levels of the protein needed to restore and maintain the clotting property of blood. For advocates, knocking out this disease in the human population makes gene therapy—despite its limitations— a worthwhile pursuit.
Gene therapy is the use of DNA as a pharmaceutical agent to treat disease. It derives its name from the idea that DNA can be used to supplement or alter genes within an individual's cells as a therapy to treat disease. The most common form of gene therapy involves using DNA that encodes a functional, therapeutic gene to replace a mutated gene. Other forms involve directly correcting a mutation, or using DNA that encodes a therapeutic protein drug (rather than a natural human gene) to provide treatment.
Gene therapy is composed of two categories: somatic gene therapy and germ line gene therapy. In somatic gene therapy, therapeutic genes are introduced to the diseased cells of a patient in hopes that they will genetically alter them to function normally. In germ line gene therapy, therapeutic genes are introduced to reproductive cells (egg and sperm cells) to prevent the manifestation of a genetic disorder before the patient is born. This approach would alter the patient’s genetic makeup and the genes he or she passes on to succeeding generations. Additionally, therapeutic genes can be introduced to cells in several ways. In ex vivo gene therapy, a patient’s