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Journal Articles

BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression

Clinical Trials & Observations
Barbara A. Konkle, Christopher E. Walsh, Miguel A. Escobar, Neil C. Josephson, Guy Young, Annette von Drygalski, Scott W. J. McPhee, R. Jude Samulski, Ivan Bilic, Maurus de la Rosa, Birgit M. Reipert, Hanspeter Rottensteiner, Friedrich Scheiflinger, John C. Chapin, Bruce Ewenstein, Paul E. Monahan
Journal: Blood
Blood (2021) 137 (6): 763–774.
https://doi.org/10.1182/blood.2019004625
Published: 2021
Includes: Supplemental data
Journal Articles

AAV8-Specific Immune Adsorption Column: A Treatment Option for Patients with Pre-Existing Anti-AAV8 Neutralizing Antibodies

Anita Kruzik, PhD, Roland Raim, PhD, Dirk Voelkel, PhD, Markus Weiller, PhD, Werner Hoellriegl, PhD, Friedrich Scheiflinger, Hanspeter Rottensteiner, Birgit M Reipert, PhD, Maurus de la Rosa
Journal: Blood
Blood (2019) 134 (Supplement_1): 5922.
https://doi.org/10.1182/blood-2019-126730
Published: 2019
Journal Articles

The Factor VIII Variant X5 Enhances Hemophilia a Gene Therapy Efficiency By Its Improved Secretion

Franziska Horling, PhD, Johannes Lengler, PhD, Wenjing Cao, PhD, Biao Dong, PhD, Bagirath Gangadharan, Maurus De La Rosa, PhD, Werner Hoellriegl, PhD, Birgit M Reipert, PhD, Friedrich Scheiflinger, Weidong Xiao, PhD, Hanspeter Rottensteiner
Journal: Blood
Blood (2019) 134 (Supplement_1): 3356.
https://doi.org/10.1182/blood-2019-126489
Published: 2019
Journal Articles

Co-Prevalence of Pre-Existing Immunity to Different Serotypes of Adeno-Associated Virus (AAV) in Adults with Hemophilia

Kavitha Rajavel, Mila Ayash-Rashkovsky, Ying Tang, Bagirath Gangadharan, Maurus de la Rosa, Bruce Ewenstein
Journal: Blood
Blood (2019) 134 (Supplement_1): 3349.
https://doi.org/10.1182/blood-2019-123666
Published: 2019
Journal Articles

CD4 + T-cell epitopes associated with antibody responses after intravenously and subcutaneously applied human FVIII in humanized hemophilic E17 HLA-DRB1*1501 mice

Katharina N. Steinitz, Pauline M. van Helden, Brigitte Binder, David C. Wraith, Sabine Unterthurner, Corinna Hermann, Maria Schuster, Rafi U. Ahmad, Markus Weiller, Christian Lubich, Maurus de la Rosa, Hans Peter Schwarz, Birgit M. Reipert
Journal: Blood
Blood (2012) 119 (17): 4073–4082.
https://doi.org/10.1182/blood-2011-08-374645
Published: 2012
Includes: Supplemental data
Journal Articles

Deciphering of CD4 + T Cell Subsets Involved in Maintenance and Break of Immune Tolerance in a Humanized Mouse Model That Is Immune Tolerant to Human FVII

Christine Lenk, Markus Pasztorek, Markus Weiller, PhD, Rafi Uddin Ahmad, PhD, Hans Peter Schwarz, MD, Friedrich Scheiflinger, PhD, Birgit M Reipert, PhD, Maurus de la Rosa, PhD
Journal: Blood
Blood (2011) 118 (21): 2188.
https://doi.org/10.1182/blood.V118.21.2188.2188
Published: 2011
Journal Articles

Induction and Break of Immune Tolerance to Human FVIII in a HLA-DRB1*1501 Humanized Hemophilic Mouse Model

Katharina Nora Steinitz, PhD, Brigitte Binder, Christian Lubich, Rafi Uddin Ahmad, PhD, Markus Weiller, PhD, Maurus de la Rosa, PhD, Hans Peter Schwarz, MD, Friedrich Scheiflinger, PhD, Birgit M Reipert, PhD
Journal: Blood
Blood (2011) 118 (21): 2280.
https://doi.org/10.1182/blood.V118.21.2280.2280
Published: 2011