Gene Therapy

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    Taxonomy Code: LT-2000

    Programs that introduce 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.

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    Service NameDescriptionLocationProgramAgency
    PRIMARY IMMUNODEFICIENCY DISEASESPediatric specialists, evaluate children with genetically defined, primary immune defects that lead to recurrent serious infection in childhood and adolescents. For these patients, bone marrow transplant or gene therapy, where appropriate, are used for immune reconstitution.NassauPRIMARY IMMUNODEFICIENCY DISEASES COHEN CHILDREN'S MEDICAL CENTER NORTHWELL HEALTH

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