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Year Number of Results
2002 1
2004 1
2007 1
2009 1
2010 1
2011 1
2012 2
2014 3
2015 3
2016 2
2017 2
2018 3
2019 3
2020 3
2022 1
2024 0

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Page 1
Fanconi anemia links reactive oxygen species to insulin resistance and obesity.
Li J, Sipple J, Maynard S, Mehta PA, Rose SR, Davies SM, Pang Q. Li J, et al. Antioxid Redox Signal. 2012 Oct 15;17(8):1083-98. doi: 10.1089/ars.2011.4417. Epub 2012 Jun 25. Antioxid Redox Signal. 2012. PMID: 22482891 Free PMC article.
Since both oxidative stress and diabetes have been observed in patients with the Fanconi anemia (FA), we sought to investigate the link between ROS and insulin resistance in this unique disease model. RESULTS: Mice deficient for the Fanconi anemia complementation …
Since both oxidative stress and diabetes have been observed in patients with the Fanconi anemia (FA), we sought to investigate the link betw …
Preclinical correction of human Fanconi anemia complementation group A bone marrow cells using a safety-modified lentiviral vector.
Becker PS, Taylor JA, Trobridge GD, Zhao X, Beard BC, Chien S, Adair J, Kohn DB, Wagner JE, Shimamura A, Kiem HP. Becker PS, et al. Gene Ther. 2010 Oct;17(10):1244-52. doi: 10.1038/gt.2010.62. Epub 2010 May 20. Gene Ther. 2010. PMID: 20485382 Free PMC article.
To minimize this damage, we have developed a brief transduction procedure for lentivirus vector-mediated transduction of hematopoietic progenitor cells from patients with Fanconi anemia complementation group A (FANCA). The lentiviral vector Fanc …
To minimize this damage, we have developed a brief transduction procedure for lentivirus vector-mediated transduction of hematopoietic proge …
Novel lineage depletion preserves autologous blood stem cells for gene therapy of Fanconi anemia complementation group A.
Adair JE, Chandrasekaran D, Sghia-Hughes G, Haworth KG, Woolfrey AE, Burroughs LM, Choi GY, Becker PS, Kiem HP. Adair JE, et al. Haematologica. 2018 Nov;103(11):1806-1814. doi: 10.3324/haematol.2018.194571. Epub 2018 Jul 5. Haematologica. 2018. PMID: 29976742 Free PMC article. Clinical Trial.
Here we demonstrate an altered ratio of CD34(Hi) to CD34(Lo) cells in Fanconi patients relative to healthy donors, with exclusive in vitro repopulating ability in only CD34(Hi) cells, underscoring a need for novel strategies to preserve limited CD34(+) cells. To address this need …
Here we demonstrate an altered ratio of CD34(Hi) to CD34(Lo) cells in Fanconi patients relative to healthy donors, with exclusive in vitro r …
Stem cell collection and gene transfer in Fanconi anemia.
Kelly PF, Radtke S, von Kalle C, Balcik B, Bohn K, Mueller R, Schuesler T, Haren M, Reeves L, Cancelas JA, Leemhuis T, Harris R, Auerbach AD, Smith FO, Davies SM, Williams DA. Kelly PF, et al. Mol Ther. 2007 Jan;15(1):211-9. doi: 10.1038/sj.mt.6300033. Mol Ther. 2007. PMID: 17164793 Free article. Clinical Trial.
Successful gene transfer into hematopoietic stem cells (HSCs) could reverse BMF in this disease. We developed clinical trials to determine whether a sufficient number of CD34(+) stem cells could be collected for gene modification and to evaluate the safety and efficacy of …
Successful gene transfer into hematopoietic stem cells (HSCs) could reverse BMF in this disease. We developed clinical trials to dete …
Minimal conditioning in Fanconi anemia promotes multi-lineage marrow engraftment at 10-fold lower cell doses.
Haworth KG, Ironside C, Ramirez MA, Weitz S, Beard BC, Schwartz JD, Adair JE, Kiem HP. Haworth KG, et al. J Gene Med. 2018 Oct;20(10-11):e3050. doi: 10.1002/jgm.3050. Epub 2018 Oct 1. J Gene Med. 2018. PMID: 30129972
RESULTS: We demonstrate that upfront low-dose conditioning greatly increases successful engraftment of hematopoietic corrected cells in a pre-clinical animal model of FA. Additionally, without conditioning, cells can still engraft and demonstrate a selective advantage in v …
RESULTS: We demonstrate that upfront low-dose conditioning greatly increases successful engraftment of hematopoietic corrected cells in a pr …
NHEJ-Mediated Repair of CRISPR-Cas9-Induced DNA Breaks Efficiently Corrects Mutations in HSPCs from Patients with Fanconi Anemia.
Román-Rodríguez FJ, Ugalde L, Álvarez L, Díez B, Ramírez MJ, Risueño C, Cortón M, Bogliolo M, Bernal S, March F, Ayuso C, Hanenberg H, Sevilla J, Rodríguez-Perales S, Torres-Ruiz R, Surrallés J, Bueren JA, Río P. Román-Rodríguez FJ, et al. Cell Stem Cell. 2019 Nov 7;25(5):607-621.e7. doi: 10.1016/j.stem.2019.08.016. Epub 2019 Sep 19. Cell Stem Cell. 2019. PMID: 31543367 Free article.
25 results