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In vivo retroviral gene transfer to ...

McCauslin, Christine Seitz.

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In vivo retroviral gene transfer to hematopoietic cells and application for phenotypic correction in a murine model of human disease.

紀錄類型:	書目-語言資料,印刷品 : Monograph/item
正題名/作者:	In vivo retroviral gene transfer to hematopoietic cells and application for phenotypic correction in a murine model of human disease./
作者:	McCauslin, Christine Seitz.
面頁冊數:	208 p.
附註:	Directors: Sally E. Spence; Jonathan R. Keller.
Contained By:	Dissertation Abstracts International62-11B.
標題:	Biology, Genetics. -
電子資源:	http://pqdd.sinica.edu.tw/twdaoapp/servlet/advanced?query=3032760
ISBN:	0493452583

In vivo retroviral gene transfer to hematopoietic cells and application for phenotypic correction in a murine model of human disease.
McCauslin, Christine Seitz.

In vivo retroviral gene transfer to hematopoietic cells and application for phenotypic correction in a murine model of human disease. - 208 p.

Directors: Sally E. Spence; Jonathan R. Keller.

Thesis (Ph.D.)--The George Washington University, 2002.

The ability to support life-long hematopoiesis makes pluripotential hematopoietic stem cells (PHSC) attractive targets for gene transfer to correct genetic disorders. However, gene transfer to PHSC has been hampered by an inability to specifically target delivery to these cells. We have investigated an approach to re-direct recombinant adeno-associated virus (rAAV) infection to PHSC in the absence of genetic vector modification. Specifically, we have re-directed rAAV infection in two different hematopoietic cell lines normally refractory to rAAV infection, KG-1a and MB-02, by covalent linkage of biotin to the viral capsid and attachment of biotinylated antibodies through an avidin bridge.

ISBN: 0493452583Subjects--Topical Terms:

1017730
Biology, Genetics.

In vivo retroviral gene transfer to hematopoietic cells and application for phenotypic correction in a murine model of human disease.
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100 1 $a McCauslin, Christine Seitz. $3 1250758
245 1 0 $a In vivo retroviral gene transfer to hematopoietic cells and application for phenotypic correction in a murine model of human disease.
300 $a 208 p.
500 $a Directors: Sally E. Spence; Jonathan R. Keller.
500 $a Source: Dissertation Abstracts International, Volume: 62-11, Section: B, page: 4918.
502 $a Thesis (Ph.D.)--The George Washington University, 2002.
520 $a The ability to support life-long hematopoiesis makes pluripotential hematopoietic stem cells (PHSC) attractive targets for gene transfer to correct genetic disorders. However, gene transfer to PHSC has been hampered by an inability to specifically target delivery to these cells. We have investigated an approach to re-direct recombinant adeno-associated virus (rAAV) infection to PHSC in the absence of genetic vector modification. Specifically, we have re-directed rAAV infection in two different hematopoietic cell lines normally refractory to rAAV infection, KG-1a and MB-02, by covalent linkage of biotin to the viral capsid and attachment of biotinylated antibodies through an avidin bridge.
520 $a Retroviruses, also commonly used as vectors for PHSC gene transfer, require that target cells undergo mitosis for proviral integration. The majority of PHSC are non-cycling; therefore, cycling is induced by <italic>ex vivo</italic> culture in cytokines that may negatively impact PHSC physiology. We have developed a murine model for <italic>in vivo</italic> retroviral gene transfer whereby retroviral particles are directly introduced into the bone marrow of the femur following cytoreductive treatment of the mice with 5-fluorouracil. Using a murine stem cell virus vector, we have observed <italic>in vivo</italic> retroviral gene transfer to both hematopoietic day 14 spleen colony-forming cells and short-term radioprotective cells at two months following treatment. Moreover, <italic> in vivo</italic> transduction of PHSC was demonstrated by the presence of transduced, mature granulocytes, B cells, and T cells 4 to 8 months following treatment. Incorporation of a selective advantage for transduced cells by direct injection of retrovirus encoding <italic>Jak3</italic> to <italic> Jak3</italic><super>−/−</super> mice with severe combined immunodeficient phenotype, lead to <italic>in vivo</italic> transduction and partial correction, both phenotypically and functionally, of cellular and humoral immune responses.
520 $a <italic>In vivo</italic> retroviral gene transfer is an attractive alternative to the current <italic>ex vivo</italic> approach because the cells are maintained in their native microenvironment with minimal disruption of endogenous signals required for maintenance of PHSC physiology. Taken together, our results suggest that direct delivery of retroviral vectors for <italic> in vivo</italic> transduction could be translated to a human clinical setting for treatment of genetic diseases including hemophilia, thalessmia, and other inborn errors of metabolism.
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