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Neonatal gene therapy. transfer and expression of exogenous genes in neonatal sheep following direct injection of retroviral vectors into the bone marrow space.

Abstract：

:We investigated whether gene transfer into hematopoietic cells could be achieved by direct injection of retroviral vector supernatant into the bone marrow space of newborn sheep. Six sheep (5 weeks old) were injected bilaterally with either 1 mL of G1nBgSvNa8.1 vector supernatant (titer: 1 x 10(7)) in each hip (n = 5) or with 3 mL of the same vector preparation/hip (n = 1). In addition, one 3-month-old sheep was injected unilaterally with 1 mL of the same vector preparation. Blood and marrow of these animals were analyzed for the transgene before injection and at intervals thereafter. At 1 week postinjection, an average of 11.6% of the lymphocytes and 25.5% of the granulocytes/monocytes in the marrow, and an average of 0.9% of the lymphocytes and 1.8% of the granulocytes/monocytes in the blood contained and expressed the LacZ gene. The presence/expression of the transgene has persisted for at least 13 months within the blood and bone marrow of these animals. These findings demonstrate that the direct injection of small volumes of high-titer retroviral supernatant into the bone marrow of newborn sheep results in transduction of hematopoietic cells that persists for at least 13 months postinjection.

journal_name

Exp Hematol

journal_title

Experimental hematology

authors

Porada CD,Tran ND,Zhao Y,Anderson WF,Zanjani ED

doi

10.1016/s0301-472x(00)00158-2

keywords:

subject

Has Abstract

pub_date

2000-06-01 00:00:00

pages

642-50

issue

6

eissn

0301-472X

issn

1873-2399

pii

S0301-472X(00)00158-2

journal_volume

28

pub_type

杂志文章

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