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Association for Research in Vision and Ophthalmology, Investigative Ophthalmology & Visual Science, 8(55), p. 5382

DOI: 10.1167/iovs.14-14179

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Monomethylfumarate Induces γ-Globin Expression and Fetal Hemoglobin Production in Cultured Human Retinal Pigment Epithelial (RPE) and Erythroid Cells, and in Intact Retina

Journal article published in 2014 by Wanwisa Promsote, Sylvia B. Smith, Biaoru Li, Levi H. Makala, Nagendra Singh, Betty S. Pace ORCID, Vadivel Ganapathy, Pamela M. Martin
This paper is made freely available by the publisher.
This paper is made freely available by the publisher.

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Abstract

Blindness in sickle cell disease is most debilitating and unfortunately common. Herein, we present for the first time, the retinal pigment epithelium as a novel target for fetal hemoglobin induction and therapeutic management of sickle cell retinopathy, and monomethylfumarate as a new and potent therapy for use in this purpose.