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David R. Lynch et al.
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Jintang Du et al.
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Elisabetta Soragni et al.
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Gabsang Lee et al.
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Stuart M. Chambers et al.
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Haiqun Jia et al.
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Maria Beconi et al.
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Naohiro Egawa et al.
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Agathi-Vasiliki Goula et al.
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Giovanni Coppola et al.
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A. F. Klein et al.
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Masayuki Nakamori et al.
HUMAN MOLECULAR GENETICS (2011)
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Maria C. Marchetto et al.
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Chiranjeevi Sandi et al.
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PHARMACEUTICALS (2011)
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Dolores Garcia-Arocena et al.
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Myriam Rai et al.
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Chunping Xu et al.
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Induced pluripotent stem cells from a spinal muscular atrophy patient
Allison D. Ebert et al.
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Scott Ditch et al.
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X. Yao et al.
BRITISH JOURNAL OF PHARMACOLOGY (2008)
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Kuanyu Li et al.
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Eriko Greene et al.
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David Herman et al.
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YF Lin et al.
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DNA triplet repeats mediate heterochromatin-protein-1-sensitive variegated gene silencing
A Saveliev et al.
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