Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency.
Publication
, Journal Article
Kohn, DB; Weinberg, KI; Nolta, JA; Heiss, LN; Lenarsky, C; Crooks, GM; Hanley, ME; Annett, G; Brooks, JS; el-Khoureiy, A
Published in: Nat Med
October 1995
Haematopoietic stem cells in umbilical cord blood are an attractive target for gene therapy of inborn errors of metabolism. Three neonates with severe combined immunodeficiency were treated by retroviral-mediated transduction of the CD34+ cells from their umbilical cord blood with a normal human adenosine deaminase complementary DNA followed by autologous transplantation. The continued presence and expression of the introduced gene in leukocytes from bone marrow and peripheral blood for 18 months demonstrates that umbilical cord blood cells may be genetically modified with retroviral vectors and engrafted in neonates for gene therapy.
Duke Scholars
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Published In
Nat Med
DOI
ISSN
1078-8956
Publication Date
October 1995
Volume
1
Issue
10
Start / End Page
1017 / 1023
Location
United States
Related Subject Headings
- Transduction, Genetic
- Severe Combined Immunodeficiency
- Retroviridae
- Molecular Sequence Data
- Male
- Lymphocyte Transfusion
- Leukocytes
- Infant, Newborn
- Immunology
- Humans
Citation
APA
Chicago
ICMJE
MLA
NLM
Kohn, D. B., Weinberg, K. I., Nolta, J. A., Heiss, L. N., Lenarsky, C., Crooks, G. M., … el-Khoureiy, A. (1995). Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency. Nat Med, 1(10), 1017–1023. https://doi.org/10.1038/nm1095-1017
Kohn, D. B., K. I. Weinberg, J. A. Nolta, L. N. Heiss, C. Lenarsky, G. M. Crooks, M. E. Hanley, G. Annett, J. S. Brooks, and A. el-Khoureiy. “Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency.” Nat Med 1, no. 10 (October 1995): 1017–23. https://doi.org/10.1038/nm1095-1017.
Kohn DB, Weinberg KI, Nolta JA, Heiss LN, Lenarsky C, Crooks GM, et al. Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency. Nat Med. 1995 Oct;1(10):1017–23.
Kohn, D. B., et al. “Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency.” Nat Med, vol. 1, no. 10, Oct. 1995, pp. 1017–23. Pubmed, doi:10.1038/nm1095-1017.
Kohn DB, Weinberg KI, Nolta JA, Heiss LN, Lenarsky C, Crooks GM, Hanley ME, Annett G, Brooks JS, el-Khoureiy A. Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency. Nat Med. 1995 Oct;1(10):1017–1023.
Published In
Nat Med
DOI
ISSN
1078-8956
Publication Date
October 1995
Volume
1
Issue
10
Start / End Page
1017 / 1023
Location
United States
Related Subject Headings
- Transduction, Genetic
- Severe Combined Immunodeficiency
- Retroviridae
- Molecular Sequence Data
- Male
- Lymphocyte Transfusion
- Leukocytes
- Infant, Newborn
- Immunology
- Humans