Adeno-Associated Virus Vectors and Hematology
Year: 1999
Type: article
Abstract: GENE THERAPISTS have a special fondness for hematol-ogy. Initially, this was due to the many inherited diseases that could potentially be cured by ex vivo genetic modification of hematopoietic stem cells. Once the early murine leukemia virus (MLV) vectors were found to transduce only a small percentage of primate stem cells, the search began for other types of vectors that might work more efficiently, and adeno-associated virus (AAV) vectors were among the first alterna-tives to be investigated. Although a substantial amount of work with AAV vectors has occurred over the last several years, the bulk of evidence suggests that they transduce hematopoietic stem cells poorly, and that they will not replace retroviral vectors in stem cell gene transfer applications. Instead, it is applications involving the production of secreted proteins that now appear especially promising for AAV vectors, where they are delivered by direct injection rather than ex vivo cell
Source: Blood
Authors: David W. Russell, Mark A. Kay
Institutions: University of Washington, Stanford University
Language: English
Subfield: Genetics
Domain: Life Sciences
Open Access status: green
OpenAlex ID: W1519039806