J. Rupon

发表

A functional screen for Krüppel-like factors that regulate the human gamma-globin gene through the CACCC promoter element.

Ping Zhang, G. D. Ginder, P. Basu, 2005, Blood cells, molecules & diseases.

Methyl binding domain protein 2 mediates gamma-globin gene silencing in adult human betaYAC transgenic mice.

G. D. Ginder, K. Gaensler, J. Rupon, 2006, Proceedings of the National Academy of Sciences of the United States of America.

Outcomes of a clinical pathway for primary outpatient management of pediatric patients with low‐risk febrile neutropenia

H. Weinstein, A. Friedmann, J. Mariani, 2019, Pediatric blood & cancer.

Presentation of hemophagocytic lymphohistiocytosis due to a novel MUNC 13–4 mutation masked by partial therapeutic immunosuppression

J. Orange, M. Mizesko, M. Paessler, 2012, Pediatric Rheumatology.

The reactions of myoglobin, normal adult hemoglobin, sickle cell hemoglobin and hemin with hydroxyurea.

S. Ballas, D. Kim‐Shapiro, S. King, 2000, Biophysical chemistry.

Moroctocog Alfa (AF-CC) for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Patients with Hemophilia A in India

S. Apte, S. Rangarajan, M. John, 2022, Indian Journal of Hematology and Blood Transfusion.

Assessment of Relative Bioavailability of Moroctocog Alfa and Moroctocog Alfa (AF‐CC) in Subjects With Severe Hemophilia A

J. Korth-Bradley, A. Płotka, P. Rendo, 2018, Clinical and translational science.

Clinical experience with moroctocog alfa (AF‐CC) in younger paediatric patients with severe haemophilia A: Two open‐label studies

J. Korth-Bradley, K. Kavaklı, J. Fuiman, 2018, Haemophilia : the official journal of the World Federation of Hemophilia.

Impact of Treatment Regimen with Moroctocog Alfa (AF-CC) on Bleeding Frequency in Pediatric Aged Subjects with Moderately Severe to Severe Hemophilia A

Mark P Smith, J. Korth-Bradley, Y. Wali, 2018, Blood.

Efficacy and Safety in 15 Hemophilia B Patients Treated with the AAV Gene Therapy Vector Fidanacogene Elaparvovec and Followed for at Least 1 Year

John E. Murphy, J. Rasko, D. Rybin, 2019, Blood.

Factor IX assay discrepancies in the setting of liver gene therapy using a hyperfunctional variant factor IX‐Padua

John E. Murphy, D. Rybin, M. Carr, 2021, Journal of thrombosis and haemostasis : JTH.

First open-label, single-arm, prospective study of real-world use of FIX replacement therapy in a predominantly pediatric hemophilia B population in China

J. Korth-Bradley, R. Wu, Lihong Xu, 2021, Medicine.

Nonacog Alfa for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Patients with Moderately Severe or Severe Hemophilia B in India

S. Apte, S. Rangarajan, M. John, 2022, Indian Journal of Hematology and Blood Transfusion.

Follow-up of More Than 5 Years in a Cohort of Patients with Hemophilia B Treated with Fidanacogene Elaparvovec Adeno-Associated Virus Gene Therapy

J. Rasko, J. Teitel, C. McGuinn, 2021, Blood.

Updated Follow-up of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (SB-525) Gene Therapy in Adults with Severe Hemophilia a

A. Leavitt, D. Rouy, B. Konkle, 2020 .

Safety and Efficacy of Moroctocog Alfa (AF-CC) in Chinese Patients with Hemophilia A: Results of Two Open-Label Studies

Jie Jin, Depei Wu, J. Korth-Bradley, 2020, Journal of blood medicine.

Iron nitrosyl hemoglobin formation from the reactions of hemoglobin and hydroxyurea.

S. Ballas, Jinming Huang, S. King, 2002, Biochemistry.

Methyl binding domain protein 2 mediates γ-globin gene silencing in adult human βYAC transgenic mice

K. Gaensler, J. Rupon, J. Lloyd, 2006 .

MBD2 contributes to developmental silencing of the human ε-globin gene.

Stefanos Labropoulos, Gordon D Ginder, Jeremy W Rupon, 2011, Blood cells, molecules & diseases.

The Role of DNA Methylation and Methyl Binding Domain Protein 2 in the Regulation of Human Embryonic and Fetal Beta Type Globin Genes

J. Rupon, 2006 .

Reactivation of Developmentally Silenced Globin Genes by Forced Chromatin Looping

Philip D. Gregory, Ann Dean, Irene Motta, 2014, Cell.

Forced chromatin looping raises fetal hemoglobin in adult sickle cells to higher levels than pharmacologic inducers.

S. Rivella, G. Blobel, Wulan Deng, 2016, Blood.

MBD2 is a critical component of a methyl cytosine-binding protein complex isolated from primary erythroid cells.

E. Kransdorf, J. Rupon, T. B. Langston, 2006, Blood.

Forced enhancer-promoter rewiring to alter gene expression in animal models

G. Blobel, B. Ryu, M. Weiss, 2023, Molecular therapy. Nucleic acids.

Using Forced Chromatin Looping To Overcome Developmental Silencing Of Embryonic and Fetal β-Type Globin Genes In Adult Erythroid Cells

P. Gregory, G. Blobel, A. Dean, 2013 .

Updated Results of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (PF-07055480/SB-525) Gene Therapy in Adults with Severe Hemophilia a

A. Leavitt, D. Rouy, B. Konkle, 2021, Blood.