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Research Article Free access | 10.1172/JCI115448

An in vivo animal model of gene therapy for leukocyte adhesion deficiency.

J C Krauss, L A Mayo-Bond, C E Rogers, K L Weber, R F Todd 3rd, and J M Wilson

Department of Internal Medicine, University of Michigan Medical School, Ann Arbor 48109-0650.

Find articles by Krauss, J. in: PubMed | Google Scholar

Department of Internal Medicine, University of Michigan Medical School, Ann Arbor 48109-0650.

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Department of Internal Medicine, University of Michigan Medical School, Ann Arbor 48109-0650.

Find articles by Rogers, C. in: PubMed | Google Scholar

Department of Internal Medicine, University of Michigan Medical School, Ann Arbor 48109-0650.

Find articles by Weber, K. in: PubMed | Google Scholar

Department of Internal Medicine, University of Michigan Medical School, Ann Arbor 48109-0650.

Find articles by Todd, R. in: PubMed | Google Scholar

Department of Internal Medicine, University of Michigan Medical School, Ann Arbor 48109-0650.

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Published October 1, 1991 - More info

Published in Volume 88, Issue 4 on October 1, 1991
J Clin Invest. 1991;88(4):1412–1417. https://doi.org/10.1172/JCI115448.
© 1991 The American Society for Clinical Investigation
Published October 1, 1991 - Version history
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Abstract

Leukocyte adhesion deficiency (LAD) is an inherited disorder of leukocyte function that is caused by defects in the CD18 gene and is associated with diminished cell surface expression of CD11/CD18 proteins. We have developed an in vivo model for gene therapy of LAD. Recombinant retroviruses were used to transduce a functional human CD18 gene into murine bone marrow cells which were transplanted into lethally irradiated syngeneic recipients. A reliable flow cytometric assay for human CD18 in transplant recipients was developed based on: (a) the availability of human specific CD18 monoclonal antibodies and (b) the observation that human CD18 can form chimeric heterodimers with murine CD11a on the cell surface. Human CD18 was detected on leukocytes in a substantial number of transplant recipients for at least 6 mo suggesting that the gene had been transduced into stem cells. Expression was demonstrated in several lineages of a variety of hematopoietic tissues, but was consistently highest and most frequent in granulocytes. Murine granulocytes demonstrated appropriate posttranscriptional regulation of human CD18 in response to activation of protein kinase C. No apparent untoward effects of human CD18 expression were noted in transplant recipients. These studies suggest a specific strategy for LAD gene therapy that may be effective and safe.

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