Literature DB >> 22248350

Gene therapy for Fanconi anemia: one step closer to the clinic.

Jakub Tolar1, Pamela S Becker, D Wade Clapp, Helmut Hanenberg, Cristina Díaz de Heredia, Hans-Peter Kiem, Susana Navarro, Pankaj Qasba, Paula Rio, Manfred Schmidt, Julián Sevilla, Els Verhoeyen, Adrian J Thrasher, Juan Bueren.   

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Year:  2012        PMID： 22248350      PMCID： PMC3277737          DOI： 10.1089/hum.2011.237

Source DB:  PubMed          Journal:  Hum Gene Ther        ISSN： 1043-0342            Impact factor:   5.695


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  29 in total

1.  Combination of HOXB4 and Delta-1 ligand improves expansion of cord blood cells.

Authors:  Korashon L Watts; Colleen Delaney; R Keith Humphries; Irwin D Bernstein; Hans-Peter Kiem
Journal:  Blood       Date:  2010-10-04       Impact factor: 22.113

2.  Stem cell collection and gene transfer in Fanconi anemia.

Authors:  Patrick F Kelly; Susan Radtke; Christof von Kalle; Brenden Balcik; Kimberley Bohn; Robin Mueller; Todd Schuesler; Moira Haren; Lilith Reeves; Jose A Cancelas; Thomas Leemhuis; Richard Harris; Arleen D Auerbach; Franklin O Smith; Stella M Davies; David A Williams
Journal:  Mol Ther       Date:  2007-01       Impact factor: 11.454

3.  Natural gene therapy in monozygotic twins with Fanconi anemia.

Authors:  Anuj Mankad; Toshiyasu Taniguchi; Barbara Cox; Yassmine Akkari; R Keaney Rathbun; Lora Lucas; Grover Bagby; Susan Olson; Alan D'Andrea; Markus Grompe
Journal:  Blood       Date:  2006-01-05       Impact factor: 22.113

4.  Gene therapy finds its niche.

Authors:  Cormac Sheridan
Journal:  Nat Biotechnol       Date:  2011-02       Impact factor: 54.908

Review 5.  Discovering pluripotency: 30 years of mouse embryonic stem cells.

Authors:  Martin Evans
Journal:  Nat Rev Mol Cell Biol       Date:  2011-09-23       Impact factor: 94.444

Review 6.  Stem cell gene therapy for fanconi anemia: report from the 1st international Fanconi anemia gene therapy working group meeting.

Authors:  Jakub Tolar; Jennifer E Adair; Michael Antoniou; Cynthia C Bartholomae; Pamela S Becker; Bruce R Blazar; Juan Bueren; Thomas Carroll; Marina Cavazzana-Calvo; D Wade Clapp; Robert Dalgleish; Anne Galy; H Bobby Gaspar; Helmut Hanenberg; Christof Von Kalle; Hans-Peter Kiem; Dirk Lindeman; Luigi Naldini; Susana Navarro; Raffaele Renella; Paula Rio; Julián Sevilla; Manfred Schmidt; Els Verhoeyen; John E Wagner; David A Williams; Adrian J Thrasher
Journal:  Mol Ther       Date:  2011-05-03       Impact factor: 11.454

7.  Treatment of Fanconi anemia patients using fludarabine and low-dose TBI, followed by unrelated donor hematopoietic cell transplantation.

Authors:  M S Thakar; P Kurre; R Storb; M Kletzel; H Frangoul; M A Pulsipher; W Leisenring; M E D Flowers; B M Sandmaier; A Woolfrey; H-P Kiem
Journal:  Bone Marrow Transplant       Date:  2010-06-28       Impact factor: 5.483

8.  AMD3100 synergizes with G-CSF to mobilize repopulating stem cells in Fanconi anemia knockout mice.

Authors:  Anna C Pulliam; M Joe Hobson; Samantha L Ciccone; Yan Li; Shi Chen; Edward F Srour; Feng-Chun Yang; Hal E Broxmeyer; D Wade Clapp
Journal:  Exp Hematol       Date:  2008-05-20       Impact factor: 3.084

9.  Nonobese diabetic/severe combined immunodeficiency (NOD/SCID) mouse as a model system to study the engraftment and mobilization of human peripheral blood stem cells.

Authors:  J C van der Loo; H Hanenberg; R J Cooper; F Y Luo; E N Lazaridis; D A Williams
Journal:  Blood       Date:  1998-10-01       Impact factor: 22.113

10.  Fanconi anemia - learning from children.

Authors:  Johanna Svahn; Carlo Dufour
Journal:  Pediatr Rep       Date:  2011-06-22
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  13 in total

Review 1.  Development of gene therapy for blood disorders: an update.

Authors:  Arthur W Nienhuis
Journal:  Blood       Date:  2013-07-10       Impact factor: 22.113

Review 2.  Multifaceted Fanconi Anemia Signaling.

Authors:  Raymond Che; Jun Zhang; Manoj Nepal; Bing Han; Peiwen Fei
Journal:  Trends Genet       Date:  2017-12-16       Impact factor: 11.639

3.  Measles virus envelope pseudotyped lentiviral vectors transduce quiescent human HSCs at an efficiency without precedent.

Authors:  Camille Lévy; Fouzia Amirache; Anais Girard-Gagnepain; Cecilia Frecha; Francisco J Roman-Rodríguez; Ornellie Bernadin; Caroline Costa; Didier Nègre; Alejandra Gutierrez-Guerrero; Lenard S Vranckx; Isabelle Clerc; Naomi Taylor; Lars Thielecke; Kerstin Cornils; Juan A Bueren; Paula Rio; Rik Gijsbers; François-Loïc Cosset; Els Verhoeyen
Journal:  Blood Adv       Date:  2017-10-24

Review 4.  Emerging cellular and gene therapies for congenital anemias.

Authors:  Leif S Ludwig; Rajiv K Khajuria; Vijay G Sankaran
Journal:  Am J Med Genet C Semin Med Genet       Date:  2016-10-28       Impact factor: 3.908

Review 5.  Stem cell-based therapies for HIV/AIDS.

Authors:  Olivier Pernet; Swati Seth Yadav; Dong Sung An
Journal:  Adv Drug Deliv Rev       Date:  2016-05-02       Impact factor: 15.470

6.  Metformin improves defective hematopoiesis and delays tumor formation in Fanconi anemia mice.

Authors:  Qing-Shuo Zhang; Weiliang Tang; Matthew Deater; Ngoc Phan; Andrea N Marcogliese; Hui Li; Muhsen Al-Dhalimy; Angela Major; Susan Olson; Raymond J Monnat; Markus Grompe
Journal:  Blood       Date:  2016-10-18       Impact factor: 22.113

7.  Improvement of genetic stability in lymphocytes from Fanconi anemia patients through the combined effect of α-lipoic acid and N-acetylcysteine.

Authors:  Filipa Ponte; Rosa Sousa; Ana Paula Fernandes; Cristina Gonçalves; José Barbot; Félix Carvalho; Beatriz Porto
Journal:  Orphanet J Rare Dis       Date:  2012-05-16       Impact factor: 4.123

Review 8.  Fanconi anemia and the cell cycle: new perspectives on aneuploidy.

Authors:  Grzegorz Nalepa; D Wade Clapp
Journal:  F1000Prime Rep       Date:  2014-04-01

9.  Long-term reproducible expression in human fetal liver hematopoietic stem cells with a UCOE-based lentiviral vector.

Authors:  Niraja Dighe; Maroun Khoury; Citra Mattar; Mark Chong; Mahesh Choolani; Jianzhu Chen; Michael N Antoniou; Jerry K Y Chan
Journal:  PLoS One       Date:  2014-08-12       Impact factor: 3.240

10.  Whole exome sequencing reveals concomitant mutations of multiple FA genes in individual Fanconi anemia patients.

Authors:  Lixian Chang; Weiping Yuan; Huimin Zeng; Quanquan Zhou; Wei Wei; Jianfeng Zhou; Miaomiao Li; Xiaomin Wang; Mingjiang Xu; Fengchun Yang; Yungui Yang; Tao Cheng; Xiaofan Zhu
Journal:  BMC Med Genomics       Date:  2014-05-15       Impact factor: 3.063
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