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Sophia C. Kamran
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Journal Articles

Optimization of Bcl11a Knockdown By miRNA Scaffold Embedded Shrnas Leading to Enhanced Induction of Fetal Hemoglobin in Erythroid Cells for the Treatment of Beta-Hemoglobinopathies

Christian Brendel, PhD, Swaroopa Guda, PhD, Raffaele Renella, MD PhD, Peng Du, PhD, Daniel E. Bauer, MD, PhD, Matthew C. Canver, BS, Sophia C. Kamran, MD, James Thornton, PhD, Helen de Boer, BAS, Michael D. Milsom, PhD, Stuart H. Orkin, MD, Richard Gregory, PhD, David A. Williams, MD
Journal: Blood
Blood (2014) 124 (21): 2150.
https://doi.org/10.1182/blood.V124.21.2150.2150
Published: 2014
Journal Articles

Fine-Mapping and Genome Editing Reveal An Essential Erythroid Enhancer At The HbF-Associated BCL11A Locus

Daniel E. Bauer, MD, PHD, Sophia C. Kamran, MD, Samuel Lessard, BS, Jian Xu, PhD, Yuko Fujiwara, PhD, Carrie Lin, BS, Zhen Shao, PhD, Matthew C. Canver, BS, Elenoe C. Smith, PhD, Luca Pinello, PhD, Peter J. Sabo, PhD, Jeff Vierstra, BS, Richard A. Voit, BS, Guo-Cheng Yuan, PhD, Matthew H. Porteus, MD, PhD, John Stamatoyannopoulos, MD, Guillaume Lettre, PhD, Stuart H. Orkin, MD
Journal: Blood
Blood (2013) 122 (21): 437.
https://doi.org/10.1182/blood.V122.21.437.437
Published: 2013
Journal Articles

HbF-Associated Genetic Variation Marks an Erythroid Regulatory Element Essential for BCL11A Transcription and Subsequent Stage-Specific Globin Expression

Daniel E. Bauer, MD, PhD, Sophia C Kamran, B.S., Jian Xu, PhD, Carrie Lin, B.S., Zhen Shao, PhD, Yuko Fujiwara, PhD, Richard A. Voit, B.S., Matthew H. Porteus, MD, PhD, John A. Stamatoyannopoulos, MD, Stuart H. Orkin, MD
Journal: Blood
Blood (2012) 120 (21): 828.
https://doi.org/10.1182/blood.V120.21.828.828
Published: 2012
Journal Articles

Reawakening fetal hemoglobin: prospects for new therapies for the β-globin disorders

Daniel E. Bauer, Sophia C. Kamran, Stuart H. Orkin
Journal: Blood
Blood (2012) 120 (15): 2945–2953.
https://doi.org/10.1182/blood-2012-06-292078
Published: 2012