Phenotypic correction of primary Fanconi anemia T cells with retroviral vectors as a diagnostic tool

Helmut Hanenberg, Karen Pollok, Karen E. Pollok, Lydia Vieten, Peter C. Verlander, Cordula Leurs, Ryan J. Cooper, Laura Haneline, D. Clapp, D. Wade Clapp, Stephan Lobitz, David A. Williams, Arleen D. Auerbach

Research output: Contribution to journalArticle

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Abstract

Objective. The aim of this study was to develop a rapid laboratory procedure that is capable of subtyping Fanconi anemia (FA) complementation groups FA-A, FA-C, FA-G, and FA-non-ACG patients from a small amount of peripheral blood. Materials and Methods. For this test, primary peripheral blood-derived FA T cells were transduced with oncoretroviral vectors that expressed FANCA, FANCC, or FANCG cDNA. We achieved a high efficiency of gene transfer into primary FA T cells by using the fibronectin fragment CH296 during transduction. Transduced cells were analyzed for correction of the characteristic DNA cross-linker hypersensitivity by cell survival or by metaphase analyses. Results. Retroviral vectors containing the cDNA for FA-A, FA-C, and FA-G, the most frequent complementation groups in North America, allowed rapid identification of the defective gene by complementation of primary T cells from 12 FA patients. Conclusion. Phenotypic correction of FA T cells using retroviral vectors can be used successfully to determine the FA complementation group immediately after diagnosis of the disease.

Original languageEnglish (US)
Pages (from-to)410-420
Number of pages11
JournalExperimental Hematology
Volume30
Issue number5
DOIs
StatePublished - 2002

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Fanconi Anemia
T-Lymphocytes
Complementary DNA
Metaphase
North America
Fibronectins
Genes

ASJC Scopus subject areas

  • Cancer Research
  • Cell Biology
  • Genetics
  • Hematology
  • Oncology
  • Transplantation

Cite this

Phenotypic correction of primary Fanconi anemia T cells with retroviral vectors as a diagnostic tool. / Hanenberg, Helmut; Pollok, Karen; Pollok, Karen E.; Vieten, Lydia; Verlander, Peter C.; Leurs, Cordula; Cooper, Ryan J.; Haneline, Laura; Clapp, D.; Clapp, D. Wade; Lobitz, Stephan; Williams, David A.; Auerbach, Arleen D.

In: Experimental Hematology, Vol. 30, No. 5, 2002, p. 410-420.

Research output: Contribution to journalArticle

Hanenberg, H, Pollok, K, Pollok, KE, Vieten, L, Verlander, PC, Leurs, C, Cooper, RJ, Haneline, L, Clapp, D, Clapp, DW, Lobitz, S, Williams, DA & Auerbach, AD 2002, 'Phenotypic correction of primary Fanconi anemia T cells with retroviral vectors as a diagnostic tool', Experimental Hematology, vol. 30, no. 5, pp. 410-420. https://doi.org/10.1016/S0301-472X(02)00782-8
Hanenberg, Helmut ; Pollok, Karen ; Pollok, Karen E. ; Vieten, Lydia ; Verlander, Peter C. ; Leurs, Cordula ; Cooper, Ryan J. ; Haneline, Laura ; Clapp, D. ; Clapp, D. Wade ; Lobitz, Stephan ; Williams, David A. ; Auerbach, Arleen D. / Phenotypic correction of primary Fanconi anemia T cells with retroviral vectors as a diagnostic tool. In: Experimental Hematology. 2002 ; Vol. 30, No. 5. pp. 410-420.
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