Next generation FIX muteins with FVIII‐independent activity for alternative treatment of hemophilia A von Quade‐Lyssy, P., Abriss, D., Milanov, P., Ungerer, C., Königs, C., Seifried, E., Schüttrumpf, J.

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Oral gene therapy for hemophilia B using chitosan‐formulated FIX mutants von Quade‐Lyssy, P., Milanov, P., Abriss, D., Ungerer, C., Königs, C., Seifried, E., Schüttrumpf, J.

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Non-Clinical Efficacy and Safety Studies on G1XCGD, a Lentiviral Vector for Ex Vivo Gene Therapy of X-Linked Chronic Granulomatous Disease von Brendel, Christian, Rothe, Michael, Santilli, Giorgia, Charrier, Sabine, Stein, Stefan, Kunkel, Hana, Abriss, Daniela, Müller-Kuller, Uta, Gaspar, Bobby, Modlich, Ute, Galy, Anne, Schambach, Axel, Thrasher, Adrian J, Grez, Manuel

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Transgene Loss and Changes in the Promoter Methylation Status as Determinants for Expression Duration in Nonviral Gene Transfer for Factor IX von SCHÜTTRUMPF, Jörg, MILANOV, Peter, ABRISS, Daniela, ROTH, Stefanie, TONN, Torsten, SEIFRIED, Erhard

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Factor IX gene therapy for the treatment of hemophilia a complicated with inhibitory antibodies von Milanov, P, Abriss, D, Tonn, T, Seifried, E, Schuettrumpf, J

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Immune response versus immune tolerance following minicircle-mediated factor VIII gene transfer von Abriss, D, Schuettrumpf, J, Milanov, P, Roth, S, Seifried, E, Tonn, T

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Non-Clinical Efficacy and Safety Studies on G1XCGD, a Lentiviral Vector for Ex Vivo Gene Therapy of X-Linked Chronic Granulomatous Disease von Brendel, C, Rothe, M, Santilli, G, Charrier, S, Stein, S, Kunkel, H, Abriss, D, Müller-Kuller, U, Gaspar, B, Modlich, U, Galy, A, Schambach, A, Thrasher, AJ, Grez, M

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Chemical chaperones improve protein secretion and rescue mutant factor VIII in mice with hemophilia A von Roth, Stefanie D, Schüttrumpf, Jörg, Milanov, Peter, Abriss, Daniela, Ungerer, Christopher, Quade-Lyssy, Patricia, Simpson, Jeremy C, Pepperkok, Rainer, Seifried, Erhard, Tonn, Torsten

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Induction of Fetal Hemoglobin and Reduction of Clinical Manifestations in Patients with Severe Sickle Cell Disease Treated with Shmir-Based Lentiviral Gene Therapy for Post-Transcr... von Esrick, Erica B., Federico, Amy, Abriss, Daniela, Armant, Myriam, Boardman, Kari, Brendel, Christian, Ciuculescu, Marioara-Felicia, Daley, Heather, Dansereau, Colleen, Fernandes, Augustine, Heeney, Matthew M., Justus, David G., Kao, Pei-Chi, Kim, Annette S., Kohn, Donald B., Lehmann, Leslie E., Liu, Donghui, London, Wendy B., Manis, John P, Moore, Theodore B., Morris, Emily, Pellin, Danilo, Proeung, Ellen, Richard, Shanna, Roach, Gavin D., Shaw, Kit L., Terrazas, Dayna, White, Shanna L, Williams, David A.

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Effects of phenylephrine on transmural distribution of myocardial blood flow in regions supplied by normal and collateral arteries during cardiopulmonary bypass von Sink, JD, Hill, RC, Chitwood, WR, Jr, Abriss, R, Wechsler, AS

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Chemical Chaperones Improve Protein Secretion and Rescue Mutant Factor VIII in Mice with Hemophilia A

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