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Triplex-forming peptide nucleic acids induce heritable elevations in gamma-globin expression in hematopoietic progenitor cells.

Chin JY, Reza F, Glazer PM.

Mol Ther. 2013 Mar;21(3):580-7. doi: 10.1038/mt.2012.262. Epub 2013 Jan 22.


Systemic delivery of triplex-forming PNA and donor DNA by nanoparticles mediates site-specific genome editing of human hematopoietic cells in vivo.

McNeer NA, Schleifman EB, Cuthbert A, Brehm M, Jackson A, Cheng C, Anandalingam K, Kumar P, Shultz LD, Greiner DL, Mark Saltzman W, Glazer PM.

Gene Ther. 2013 Jun;20(6):658-69. doi: 10.1038/gt.2012.82. Epub 2012 Oct 18.


The new self-inactivating lentiviral vector for thalassemia gene therapy combining two HPFH activating elements corrects human thalassemic hematopoietic stem cells.

Papanikolaou E, Georgomanoli M, Stamateris E, Panetsos F, Karagiorga M, Tsaftaridis P, Graphakos S, Anagnou NP.

Hum Gene Ther. 2012 Jan;23(1):15-31. doi: 10.1089/hum.2011.048. Epub 2011 Dec 5.


Nanoparticles deliver triplex-forming PNAs for site-specific genomic recombination in CD34+ human hematopoietic progenitors.

McNeer NA, Chin JY, Schleifman EB, Fields RJ, Glazer PM, Saltzman WM.

Mol Ther. 2011 Jan;19(1):172-80. doi: 10.1038/mt.2010.200. Epub 2010 Sep 21.


Mi2β is required for γ-globin gene silencing: temporal assembly of a GATA-1-FOG-1-Mi2 repressor complex in β-YAC transgenic mice.

Costa FC, Fedosyuk H, Chazelle AM, Neades RY, Peterson KR.

PLoS Genet. 2012;8(12):e1003155. doi: 10.1371/journal.pgen.1003155. Epub 2012 Dec 20.


Correction of a splice-site mutation in the beta-globin gene stimulated by triplex-forming peptide nucleic acids.

Chin JY, Kuan JY, Lonkar PS, Krause DS, Seidman MM, Peterson KR, Nielsen PE, Kole R, Glazer PM.

Proc Natl Acad Sci U S A. 2008 Sep 9;105(36):13514-9. doi: 10.1073/pnas.0711793105. Epub 2008 Aug 29.


Mi2β-mediated silencing of the fetal γ-globin gene in adult erythroid cells.

Amaya M, Desai M, Gnanapragasam MN, Wang SZ, Zu Zhu S, Williams DC Jr, Ginder GD.

Blood. 2013 Apr 25;121(17):3493-501. doi: 10.1182/blood-2012-11-466227. Epub 2013 Feb 26.


A cell-based high-throughput screen for novel chemical inducers of fetal hemoglobin for treatment of hemoglobinopathies.

Peterson KR, Costa FC, Fedosyuk H, Neades RY, Chazelle AM, Zelenchuk L, Fonteles AH, Dalal P, Roy A, Chaguturu R, Li B, Pace BS.

PLoS One. 2014 Sep 16;9(9):e107006. doi: 10.1371/journal.pone.0107006. eCollection 2014.


A single point mutation is the cause of the Greek form of hereditary persistence of fetal haemoglobin.

Berry M, Grosveld F, Dillon N.

Nature. 1992 Aug 6;358(6386):499-502.


Sequences located 3' to the breakpoint of the hereditary persistence of fetal hemoglobin-3 deletion exhibit enhancer activity and can modify the developmental expression of the human fetal A gamma-globin gene in transgenic mice.

Anagnou NP, Perez-Stable C, Gelinas R, Costantini F, Liapaki K, Constantopoulou M, Kosteas T, Moschonas NK, Stamatoyannopoulos G.

J Biol Chem. 1995 Apr 28;270(17):10256-63.


Peptide nucleic acid (PNA) binding-mediated induction of human gamma-globin gene expression.

Wang G, Xu X, Pace B, Dean DA, Glazer PM, Chan P, Goodman SR, Shokolenko I.

Nucleic Acids Res. 1999 Jul 1;27(13):2806-13.


Eos negatively regulates human γ-globin gene transcription during erythroid differentiation.

Yu HC, Zhao HL, Wu ZK, Zhang JW.

PLoS One. 2011;6(7):e22907. doi: 10.1371/journal.pone.0022907. Epub 2011 Jul 28.


Molecular analysis of the human fetal-to-adult globin switching.

Katsube T, Fucharoen S, Tojo H, Fukumaki Y.

Southeast Asian J Trop Med Public Health. 1995;26 Suppl 1:212-20.


alpha-Amino butyric acid cannot reactivate the silenced gamma gene of the beta locus YAC transgenic mouse.

Pace B, Li Q, Peterson K, Stamatoyannopoulos G.

Blood. 1994 Dec 15;84(12):4344-53.


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