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Proceedings of the National Academy of Sciences of the United States of America|November 12, 2009
Reprogramming erythroid cells for lysosomal enzyme production leads to visceral and CNS cross-correction in mice with Hurler syndromeDaren Wang, Wei Zhang, Theodosia A Kalfa, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|December 14, 2006
Murine retroviral but not human cellular promoters induce in vivo erythroid-specific deregulation that can be partially prevented by insulatorsElodie Robert-Richard, Emmanuel Richard, Punam Malik, et al.
Nature Communications|June 30, 2019
CRISPR-Cas9 fusion to dominant-negative 53BP1 enhances HDR and inhibits NHEJ specifically at Cas9 target sitesRajeswari Jayavaradhan, Devin M Pillis, Michael Goodman, et al.
Plos One|October 3, 2012
Stem cell selection in vivo using foamy vectors cures canine pyruvate kinase deficiencyGrant D Trobridge, Brian C Beard, Robert A Wu, et al.
American Journal of Hematology|March 9, 2023
Longitudinal effect of disease-modifying therapy on left ventricular diastolic function in children with sickle cell anemiaParul Rai, Victoria I Okhomina, Guolian Kang, et al.
Journal of Surgical Education|November 6, 2025
Advancing Future Surgeons: Utilization of Surgical Simulation in Medical Student Surgery ClerkshipsBenjamin Thomae, Parul Rai, Kristof S Gutowski, et al.
Human Gene Therapy|July 30, 2016
High Level of Perforin Expression Is Required for Effective Correction of Hemophagocytic LymphohistiocytosisSwati Tiwari, Adrianne Hontz, Catherine E Terrell, et al.
Molecular and Cellular Biology|November 19, 2014
MicroRNA 648 Targets ET-1 mRNA and is cotranscriptionally regulated with MICAL3 by PAX5Chen Li, Caryn S Gonsalves, Marthe-Sandrine Eiymo Mwa Mpollo, et al.
Angiogenesis|April 8, 2022
NRASQ61R mutation in human endothelial cells causes vascular malformationsElisa Boscolo, Patricia Pastura, Sandra Schrenk, et al.
The Journal of Biological Chemistry|November 13, 2014
Peroxisome proliferator-activated receptor-α-mediated transcription of miR-199a2 attenuates endothelin-1 expression via hypoxia-inducible factor-1αChen Li, Marthe-Sandrine Eiymo Mwa Mpollo, Caryn S Gonsalves, et al.
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