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Functional correction of fanconi anemia group C hematopoietic cells by the use of a novel lentiviral vector.
Mol Ther. 2001 Apr; 3(4):485-90.MT

Abstract

Lentiviral vectors transduce nondividing hematopoietic cells more efficiently than other currently available vector systems. Here we report the results of human hematopoietic cell gene transfer using lentiviral vectors based upon human immunodeficiency virus (HIV-1) and equine infectious anemia virus (EIAV). EIAV is a nonprimate lentivirus and is severely restricted in its host range to horses and closely related equines. We employed the EIAV vector system to test for gene transfer to human Fanconi anemia (FA) hematopoietic cells by comparison with HIV-1- and Moloney murine leukemia virus-based systems. Fanconi anemia is characterized by bone marrow failure secondary to stem cell dysfunction. Fanconi anemia group C EBV-transformed lymphoblasts were transduced with all three viral vectors. Phenotypic correction of FA cells, as measured by mitomycin C drug resistance, was observed with a similar efficiency in all vector systems. This is the first description of lentiviral correction of FA cells and suggests that lentiviral vectors may be useful for FA gene transfer.

Authors+Show Affiliations

UNC Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina 27599, USA.No affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Journal Article
Research Support, Non-U.S. Gov't

Language

eng

PubMed ID

11319908

Citation

Yamada, K, et al. "Functional Correction of Fanconi Anemia Group C Hematopoietic Cells By the Use of a Novel Lentiviral Vector." Molecular Therapy : the Journal of the American Society of Gene Therapy, vol. 3, no. 4, 2001, pp. 485-90.
Yamada K, Olsen JC, Patel M, et al. Functional correction of fanconi anemia group C hematopoietic cells by the use of a novel lentiviral vector. Mol Ther. 2001;3(4):485-90.
Yamada, K., Olsen, J. C., Patel, M., Rao, K. W., & Walsh, C. E. (2001). Functional correction of fanconi anemia group C hematopoietic cells by the use of a novel lentiviral vector. Molecular Therapy : the Journal of the American Society of Gene Therapy, 3(4), 485-90.
Yamada K, et al. Functional Correction of Fanconi Anemia Group C Hematopoietic Cells By the Use of a Novel Lentiviral Vector. Mol Ther. 2001;3(4):485-90. PubMed PMID: 11319908.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Functional correction of fanconi anemia group C hematopoietic cells by the use of a novel lentiviral vector. AU - Yamada,K, AU - Olsen,J C, AU - Patel,M, AU - Rao,K W, AU - Walsh,C E, PY - 2001/4/26/pubmed PY - 2001/7/28/medline PY - 2001/4/26/entrez SP - 485 EP - 90 JF - Molecular therapy : the journal of the American Society of Gene Therapy JO - Mol Ther VL - 3 IS - 4 N2 - Lentiviral vectors transduce nondividing hematopoietic cells more efficiently than other currently available vector systems. Here we report the results of human hematopoietic cell gene transfer using lentiviral vectors based upon human immunodeficiency virus (HIV-1) and equine infectious anemia virus (EIAV). EIAV is a nonprimate lentivirus and is severely restricted in its host range to horses and closely related equines. We employed the EIAV vector system to test for gene transfer to human Fanconi anemia (FA) hematopoietic cells by comparison with HIV-1- and Moloney murine leukemia virus-based systems. Fanconi anemia is characterized by bone marrow failure secondary to stem cell dysfunction. Fanconi anemia group C EBV-transformed lymphoblasts were transduced with all three viral vectors. Phenotypic correction of FA cells, as measured by mitomycin C drug resistance, was observed with a similar efficiency in all vector systems. This is the first description of lentiviral correction of FA cells and suggests that lentiviral vectors may be useful for FA gene transfer. SN - 1525-0016 UR - https://www.unboundmedicine.com/medline/citation/11319908/Functional_correction_of_fanconi_anemia_group_C_hematopoietic_cells_by_the_use_of_a_novel_lentiviral_vector_ L2 - https://linkinghub.elsevier.com/retrieve/pii/S1525-0016(01)90287-4 DB - PRIME DP - Unbound Medicine ER -