Creative a Selective Advantage for Stem Cells: A Strategy for Gene Therapy

Hdl Handle:
http://hdl.handle.net/10675.2/550469
Title:
Creative a Selective Advantage for Stem Cells: A Strategy for Gene Therapy
Authors:
Menezes, Kareena M.
Abstract:
(Statement of the Problem) Various approaches are used to treat the many known genetic diseases. The treatments are often incompletely effective, and they sometimes have undesirable side effects. Somatic cell gene therapy might provide truly effective permanent cures. Gene therapy, however, is still in the experimental stages, and much needs to be learned about stem cell biology before gene therapy becomes routine clinical practice. Moreover, inferences made from experiments in vitro do not necessarily model the in vitro setting. If treatments designed and tested in vitro can also be made workable and proven to be therapeutic in vivo, a major contribution to clinical gene therapy would be achieved. The described research, which attempts to encourage the stem cells to proliferate rather than divide down the hematopoietic cascade, could be significant in terms of increasing in number those hematopoietic cells that have been successfully modified by therapeutic vectors. The long-term goal of this research is to find a way to provide modified stem cells with a selective advantage in repopulating the marrow of a patient with a genetic disease. Ultimately it will be necessary to confer the selective advantage on somatic cells by introducing DNA into the patient’s defective bone marrow stem cells. However for purposes of preliminary laboratory analyses, a more reproducible system of testing a candidate genes’ potential for providing a selective advantage is necessary. In the present case, an Erythropoietin Receptor transgenic mouse line is used to provide stem cells, each of which already expresses the candidate selective-advantage gene.
Affiliation:
Not Listed
Issue Date:
Oct-1999
URI:
http://hdl.handle.net/10675.2/550469
Additional Links:
http://ezproxy.gru.edu/login?url=http://search.proquest.com/docview/304539189?accountid=12365
Type:
Dissertation
Appears in Collections:
Theses and Dissertations

Full metadata record

DC FieldValue Language
dc.contributor.authorMenezes, Kareena M.en
dc.date.accessioned2015-04-23T02:44:32Zen
dc.date.available2015-04-23T02:44:32Zen
dc.date.issued1999-10en
dc.identifier.urihttp://hdl.handle.net/10675.2/550469en
dc.description.abstract(Statement of the Problem) Various approaches are used to treat the many known genetic diseases. The treatments are often incompletely effective, and they sometimes have undesirable side effects. Somatic cell gene therapy might provide truly effective permanent cures. Gene therapy, however, is still in the experimental stages, and much needs to be learned about stem cell biology before gene therapy becomes routine clinical practice. Moreover, inferences made from experiments in vitro do not necessarily model the in vitro setting. If treatments designed and tested in vitro can also be made workable and proven to be therapeutic in vivo, a major contribution to clinical gene therapy would be achieved. The described research, which attempts to encourage the stem cells to proliferate rather than divide down the hematopoietic cascade, could be significant in terms of increasing in number those hematopoietic cells that have been successfully modified by therapeutic vectors. The long-term goal of this research is to find a way to provide modified stem cells with a selective advantage in repopulating the marrow of a patient with a genetic disease. Ultimately it will be necessary to confer the selective advantage on somatic cells by introducing DNA into the patient’s defective bone marrow stem cells. However for purposes of preliminary laboratory analyses, a more reproducible system of testing a candidate genes’ potential for providing a selective advantage is necessary. In the present case, an Erythropoietin Receptor transgenic mouse line is used to provide stem cells, each of which already expresses the candidate selective-advantage gene.en
dc.relation.urlhttp://ezproxy.gru.edu/login?url=http://search.proquest.com/docview/304539189?accountid=12365en
dc.rightsCopyright protected. Unauthorized reproduction or use beyond the exceptions granted by the Fair Use clause of U.S. Copyright law may violate federal law.en
dc.subjectin vitroen
dc.subjectin vivoen
dc.subjectGene Therapyen
dc.titleCreative a Selective Advantage for Stem Cells: A Strategy for Gene Therapyen
dc.typeDissertationen
dc.contributor.departmentNot Listeden
dc.description.advisorWhitney, Barryen
dc.description.committeeHoward, Eugene; Munn, David; Scott, David; Lanclos, Kennethen
dc.description.degreeDoctor of Philosophy (Ph.D.)en
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