Literature DB >> 12031647

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

Helmut Hanenberg1, Sat Dev Batish, Karen E Pollok, Lydia Vieten, Peter C Verlander, Cordula Leurs, Ryan J Cooper, Kerstin Göttsche, Laura Haneline, D Wade Clapp, Stephan Lobitz, David A Williams, Arleen D Auerbach.   

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-nonACG 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.

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Year:  2002        PMID: 12031647     DOI: 10.1016/s0301-472x(02)00782-8

Source DB:  PubMed          Journal:  Exp Hematol        ISSN: 0301-472X            Impact factor:   3.084


  26 in total

Review 1.  Fanconi anaemia.

Authors:  M D Tischkowitz; S V Hodgson
Journal:  J Med Genet       Date:  2003-01       Impact factor: 6.318

2.  Fanconi anemia with sun-sensitivity caused by a Xeroderma pigmentosum-associated missense mutation in XPF.

Authors:  Isabell Popp; Maqsood Punekar; Nick Telford; Stavros Stivaros; Kate Chandler; Meenakshi Minnis; Anna Castleton; Claire Higham; Louise Hopewell; D Gareth Evans; Anja Raams; Arjan F Theil; Stefan Meyer; Detlev Schindler
Journal:  BMC Med Genet       Date:  2018-01-11       Impact factor: 2.103

3.  Evaluation of different protocols for gene transfer into non-obese diabetes/severe combined immunodeficiency disease mouse repopulating cells.

Authors:  Peter Ebeling; P Bach; U Sorg; A Schneider; T Trarbach; D Dilloo; H Hanenberg; S Niesert; S Seeber; T Moritz; M Flasshove
Journal:  J Cancer Res Clin Oncol       Date:  2006-10-20       Impact factor: 4.553

4.  Correct mRNA processing at a mutant TT splice donor in FANCC ameliorates the clinical phenotype in patients and is enhanced by delivery of suppressor U1 snRNAs.

Authors:  Linda Hartmann; Kornelia Neveling; Stephanie Borkens; Hildegard Schneider; Marcel Freund; Elke Grassman; Stephan Theiss; Angela Wawer; Stefan Burdach; Arleen D Auerbach; Detlev Schindler; Helmut Hanenberg; Heiner Schaal
Journal:  Am J Hum Genet       Date:  2010-10-08       Impact factor: 11.025

5.  Hypomorphic mutations in the gene encoding a key Fanconi anemia protein, FANCD2, sustain a significant group of FA-D2 patients with severe phenotype.

Authors:  Reinhard Kalb; Kornelia Neveling; Holger Hoehn; Hildegard Schneider; Yvonne Linka; Sat Dev Batish; Curtis Hunt; Marianne Berwick; Elsa Callen; Jordi Surralles; Jose A Casado; Juan Bueren; Angeles Dasi; Jean Soulier; Eliane Gluckman; C Michel Zwaan; Rosalina van Spaendonk; Gerard Pals; Johan P de Winter; Hans Joenje; Markus Grompe; Arleen D Auerbach; Helmut Hanenberg; Detlev Schindler
Journal:  Am J Hum Genet       Date:  2007-04-06       Impact factor: 11.025

6.  Mutation of the RAD51C gene in a Fanconi anemia-like disorder.

Authors:  Fiona Vaz; Helmut Hanenberg; Beatrice Schuster; Karen Barker; Constanze Wiek; Verena Erven; Kornelia Neveling; Daniela Endt; Ian Kesterton; Flavia Autore; Franca Fraternali; Marcel Freund; Linda Hartmann; David Grimwade; Roland G Roberts; Heiner Schaal; Shehla Mohammed; Nazneen Rahman; Detlev Schindler; Christopher G Mathew
Journal:  Nat Genet       Date:  2010-04-18       Impact factor: 38.330

7.  Germline mutations in breast and ovarian cancer pedigrees establish RAD51C as a human cancer susceptibility gene.

Authors:  Alfons Meindl; Heide Hellebrand; Constanze Wiek; Verena Erven; Barbara Wappenschmidt; Dieter Niederacher; Marcel Freund; Peter Lichtner; Linda Hartmann; Heiner Schaal; Juliane Ramser; Ellen Honisch; Christian Kubisch; Hans E Wichmann; Karin Kast; Helmut Deissler; Christoph Engel; Bertram Müller-Myhsok; Kornelia Neveling; Marion Kiechle; Christopher G Mathew; Detlev Schindler; Rita K Schmutzler; Helmut Hanenberg
Journal:  Nat Genet       Date:  2010-04-18       Impact factor: 38.330

8.  Mesenchymal stem/progenitor cells promote the reconstitution of exogenous hematopoietic stem cells in Fancg-/- mice in vivo.

Authors:  Yan Li; Shi Chen; Jin Yuan; Yanzhu Yang; Jingling Li; Jin Ma; Xiaohua Wu; Marcel Freund; Karen Pollok; Helmut Hanenberg; W Scott Goebel; Feng-Chun Yang
Journal:  Blood       Date:  2009-01-07       Impact factor: 22.113

9.  Genetic heterogeneity among Fanconi anemia heterozygotes and risk of cancer.

Authors:  Marianne Berwick; Jaya M Satagopan; Leah Ben-Porat; Ann Carlson; Katherine Mah; Rashida Henry; Raffaella Diotti; Kelly Milton; Kanan Pujara; Tom Landers; Sat Dev Batish; José Morales; Detlev Schindler; Helmut Hanenberg; Robert Hromas; Orna Levran; Arleen D Auerbach
Journal:  Cancer Res       Date:  2007-10-01       Impact factor: 12.701

10.  Fanconi anemia deficiency stimulates HPV-associated hyperplastic growth in organotypic epithelial raft culture.

Authors:  E E Hoskins; T A Morris; J M Higginbotham; N Spardy; E Cha; P Kelly; D A Williams; K A Wikenheiser-Brokamp; S Duensing; S I Wells
Journal:  Oncogene       Date:  2008-11-17       Impact factor: 9.867

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