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Gene therapy for hemophilia B using CB 2679d-GT: a novel factor IX variant with higher potency than factor IX Padua

Brief Report
Nisha Nair, Dries De Wolf, Phuong Anh Nguyen, Quang Hong Pham, Ermira Samara-Kuko, Jeff Landau, Grant E. Blouse, Marinee K. Chuah, Thierry VandenDriessche
Journal: Blood
Blood (2021) 137 (21): 2902–2906.
https://doi.org/10.1182/blood.2020006005
Published: 2021
Includes: Supplemental data
Journal Articles

The Complete Dependence of Factor IX Padua (R338L) Hyperactivity on Factor VIIIa Cofactor Activity Supports Its Safety As a Transgene for Hemophilia B Gene Therapy

Benjamin J. Samelson-Jones, MD PhD, John D. Finn, PhD, Rodney M. Camire, PhD, Valder R Arruda, MD PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 3486.
https://doi.org/10.1182/blood-2018-99-119270
Published: 2018
Journal Articles

Adeno-Associated Virus Mediated Gene Transfer with Hyperfunctional FIX-E410H Variant Improves Mean Factor IX Activity Levels in Hemophilia B Mice

Sandra Le Quellec, MD, Allison P Dane, PhD, Claude Negrier, Yesim Dargaud, MD PhD, Amit C Nathwani, MD PhD, Nathalie Enjolras, PhD
Journal: Blood
Blood (2017) 130 (Supplement 1): 4610.
https://doi.org/10.1182/blood.V130.Suppl_1.4610.4610
Published: 2017
Journal Articles

Factor IX Padua: From Biochemistry to Gene Therapy

Valder Arruda, MD PhD, Ben J. Samelson-Jones, MDPhD
Journal: Blood
Blood (2016) 128 (22): SCI-9.
https://doi.org/10.1182/blood.V128.22.SCI-9.SCI-9
Published: 2016
Journal Articles

New Hyperactive Factor IX Variants Bioengineered Based upon Biochemical Studies of Factor IX Padua

Ben J. Samelson-Jones, MD PhD, Jonathan D Finn, PhD, Rodney M. Camire, PhD, Valder R Arruda, MD PhD
Journal: Blood
Blood (2016) 128 (22): 1384.
https://doi.org/10.1182/blood.V128.22.1384.1384
Published: 2016
Journal Articles

Factor IX Padua: them that have, give

Jay N. Lozier
Journal: Blood
Blood (2012) 120 (23): 4452–4453.
https://doi.org/10.1182/blood-2012-09-452821
Published: 2012
Journal Articles

Prevalence of FACTOR IX-R338L (FACTOR IX PADUA) IN A COHORT of PATIENTS with Venous Thromboembolism and Mild Elevation of FACTOR IX LEVELS.

Bruna de Moraes Mazetto, Fernanda A. Orsi, MD, Lúcia Helena Siqueira, Erich Vinicius de Paula, MD, PhD, Tayana B Mello, Joyce Maria Annichino-Bizzacchi, MD, PhD
Journal: Blood
Blood (2009) 114 (22): 5057.
https://doi.org/10.1182/blood.V114.22.5057.5057
Published: 2009