Adeno-associated virus vectors for gene therapy of the hematopoietic system.

Abstract

Over the past 30 years, bone marrow transplantation has come to be accepted as standard curative therapy for many pathophysiologic conditions (Forman et al. 1994) including malignancies (Brenner et al. 1993) and metabolic diseases (Blaese 1993). Thus, the reconstitution of the hematopoietic system with normal allogeneic cells capable of providing long term engraftment with disease-free, physiologically functional cells has been used to treat various inherited diseases including severe combined immunodeficiency disease (SCID), lysosomal storage disorders and various hemoglobinopathies. However, difficulties in acquisition of histocompatible marrow cells for transplantation and onset of graft vs host disease has led to the continued search for better therapeutic strategies. Meanwhile, the delineation of the genetic bases of many inherited diseases and the subsequent isolation of appropriate wild-type genes promoted the feasibility of disease correction at the DNA level. To this end, much effort has focussed upon the development of both the identification of genetic strategies as well as efficient methods of delivery of therapeutic genes to appropriate target cells.KeywordsHematopoietic Progenitor CellRous Sarcoma VirusSevere Combine Immunodeficiency DiseaseNondividing CellAdenosine Deaminase DeficiencyThese keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.