Comparative analysis of FV vectors with human α- or β-globin gene regulatory elements for the correction of β-thalassemia
Author:
Publisher
Springer Science and Business Media LLC
Subject
Genetics,Molecular Biology,Molecular Medicine
Link
http://www.nature.com/articles/gt201198.pdf
Reference42 articles.
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2. Grosveld F, van Assendelft GB, Greaves DR, Kollias G . Position-independent, high-level expression of the human beta-globin gene in transgenic mice. Cell 1987; 51: 975–985.
3. Novak U, Harris EA, Forrester W, Groudine M, Gelinas R . High-level beta-globin expression after retroviral transfer of locus activation region-containing human beta-globin gene derivatives into murine erythroleukemia cells. Proc Natl Acad Sci USA 1990; 87: 3386–3390.
4. Miccio A, Cesari R, Lotti F, Rossi C, Sanvito F, Ponzoni M et al. In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of beta-thalassemia. Proc Natl Acad Sci USA 2008; 105: 10547–10552.
5. Roselli EA, Mezzadra R, Frittoli MC, Maruggi G, Biral E, Mavilio F et al. Correction of beta-thalassemia major by gene transfer in haematopoietic progenitors of pediatric patients. EMBO Mol Med 2010; 2: 315–328.
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