Gene therapy: alteration of somatic or germ-line DNA to correct or prevent disease; the process of inserting a gene artificially into the genome of an organism to correct a genetic defect or to add a new biologic property or function with therapeutic potential.In somatic gene therapy, functional DNA sequences are inserted into cells that lack a specific gene or bear a faulty version of it. Vectors include replication-defective viruses, liposomes, and plasmids. For transfer of genetic material by viral infection (called transduction), retroviruses are particularly suitable as vectors because their RNA, converted to DNA by reverse transcriptase, becomes part of the genome of the infected cell. Adenovirus and herpesvirus are also used. Progress has been made in treating several inherited disorders, including severe combined immunodeficiency, cystic fibrosis, and hemophilia B. Gene therapy has several applications in oncology, including the transduction into malignant tumor cells of genes encoding cytokines or coactivation factors to augment host antitumor responses and the transfer of tumor suppressor genes, particularly p53 (the most commonly mutated gene found in human cancers), to enhance the sensitivity of malignant cells to chemotherapeutic agents. Use of viral vectors is associated with a risk of localized and systemic inflammation mediated by cytokines, which can be fatal. Germ-line therapy inserts specific genes directly into the DNA of sperm, egg, or embryo, producing heritable alterations of the genome.
Source: Stedman's Medical Spellchecker, © 2006 Lippincott Williams & Wilkins. All rights reserved.
Gene therapy: introduction of functioning gene or genes into cells for the purpose of correcting an inborn genetic error, treating a disease by restoring or adding gene expression, or providing a new function to the cell; new genes may be introduced into proliferating cells in vivo (e.g., bone marrow) or in vitro (e.g., fibroblast cultures) and the modified cells transferred to the site where the gene expression is required.
Source: CRISP
Gene therapy: The introduction of new genes into cells for the purpose of treating disease by restoring or adding gene expression. Techniques include insertion of retroviral vectors, transfection, homologous recombination, and injection of new genes into the nuclei of single cell embryos. The entire gene therapy process may consist of multiple steps. The new genes may be introduced into proliferating cells in vivo (e.g., bone marrow) or in vitro (e.g., fibroblast cultures) and the modified cells transferred to the site where the gene expression is required. Gene therapy may be particularly useful for treating enzyme deficiency diseases, hemoglobinopathies, and leukemias and may also prove useful in restoring drug sensitivity, particularly for leukemia.
Source: MeSH 2007
These medical condition or symptom topics may be relevant to medical information for Gene therapy:
Source - MeSH 2007
Source - MeSH 2007
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Source - MeSH 2007
Source - CRISP
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The following list attempts to classify Gene therapy into categories where each line is subset of the next.
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