A novel strategy for the screening for platelet refractoriness: prospects and limitations

por Arruda, Valder R.
Publicado 2013

Why is AAV FVIII gene therapy not approved by the US Food and Drug Administration yet?

por Arruda, Valder R.
Publicado 2021

Gene Therapy for Hemophilia: Facts and Quandaries in the 21st Century

por Arruda, Valder R., Doshi, Bhavya S.
Publicado 2020

Protein-Engineered Coagulation Factors for Hemophilia Gene Therapy

por Samelson-Jones, Benjamin J., Arruda, Valder R.
Publicado 2018

Translational Potential of Immune Tolerance Induction by AAV Liver-Directed Factor VIII Gene Therapy for Hemophilia A

por Samelson-Jones, Benjamin J., Arruda, Valder R.
Publicado 2020

Immune complications and their management in inherited and acquired bleeding disorders

por Arruda, Valder R., Lillicrap, David, Herzog, Roland W.
Publicado 2022

Emerging therapies for hemophilia: controversies and unanswered questions

por Arruda, Valder R., Doshi, Bhavya S., Samelson-Jones, Benjamin J.
Publicado 2018

Timing of Intensive Immunosuppression Impacts Risk of Transgene Antibodies after AAV Gene Therapy in Nonhuman Primates

por Samelson-Jones, Benjamin J., Finn, Jonathan D., Favaro, Patricia, Wright, J. Fraser, Arruda, Valder R.
Publicado 2020

Successful Phenotype Improvement following Gene Therapy for Severe Hemophilia A in Privately Owned Dogs

por Callan, Mary Beth, Haskins, Mark E., Wang, Ping, Zhou, Shangzhen, High, Katherine A., Arruda, Valder R.
Publicado 2016

B cell–activating factor modulates the factor VIII immune response in hemophilia A

por Doshi, Bhavya S., Rana, Jyoti, Castaman, Giancarlo, Shaheen, Mostafa A., Kaczmarek, Radoslaw, Butterfield, John S.S., Meeks, Shannon L., Leissinger, Cindy, Biswas, Moanaro, Arruda, Valder R.
Publicado 2021

Characterization of Naturally-Occurring Humoral Immunity to AAV in Sheep

por Tellez, Joseph, Van Vliet, Kim, Tseng, Yu-Shan, Finn, Jonathan D., Tschernia, Nick, Almeida-Porada, Graça, Arruda, Valder R., Agbandje-McKenna, Mavis, Porada, Christopher D.
Publicado 2013

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

por Robinson, Mary M., George, Lindsey A., Carr, Marcus E., Samelson‐Jones, Benjamin J., Arruda, Valder R., Murphy, John E., Rybin, Denis, Rupon, Jeremy, High, Katherine A., Tiefenbacher, Stefan
Publicado 2021

Correction of the coagulation defect in hemophilia using a factor Xa variant with novel engineered protease function

por Ivanciu, Lacramioara, Toso, Raffaella, Margaritis, Paris, Pavani, Giulia, Kim, Haein, Schlachterman, Alexander, Liu, Jian-Hua, Clerin, Valerie, Pittman, Debra D., Rose-Miranda, Rosalind, Shields, Kathleen M., Erbe, David V., Tobin, James F., Arruda, Valder R., Camire, Rodney M.
Publicado 2011

Safety of AAV Factor IX Peripheral Transvenular Gene Delivery to Muscle in Hemophilia B Dogs

por Haurigot, Virginia, Mingozzi, Federico, Buchlis, George, Hui, Daniel J, Chen, Yifeng, Basner-Tschakarjan, Etiena, Arruda, Valder R, Radu, Antoneta, Franck, Helen G, Wright, J Fraser, Zhou, Shangzhen, Stedman, Hansell H, Bellinger, Dwight A, Nichols, Timothy C, High, Katherine A
Publicado 2010