Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease von Esrick, Erica B, Lehmann, Leslie E, Biffi, Alessandra, Achebe, Maureen, Brendel, Christian, Ciuculescu, Marioara F, Daley, Heather, MacKinnon, Brenda, Morris, Emily, Federico, Amy, Abriss, Daniela, Boardman, Kari, Khelladi, Radia, Shaw, Kit, Negre, Helene, Negre, Olivier, Nikiforow, Sarah, Ritz, Jerome, Pai, Sung-Yun, London, Wendy B, Dansereau, Colleen, Heeney, Matthew M, Armant, Myriam, Manis, John P, Williams, David A

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Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown for Sickle Cell Gene Therapy von Brendel, Christian, Negre, Olivier, Rothe, Michael, Guda, Swaroopa, Parsons, Geoff, Harris, Chad, McGuinness, Meaghan, Abriss, Daniela, Tsytsykova, Alla, Klatt, Denise, Bentler, Martin, Pellin, Danilo, Christiansen, Lauryn, Schambach, Axel, Manis, John, Trebeden-Negre, Helene, Bonner, Melissa, Esrick, Erica, Veres, Gabor, Armant, Myriam, Williams, David A.

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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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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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Oxygen-induced changes in hemoglobin expression in Drosophila von Gleixner, Eva, Abriss, Daniela, Adryan, Boris, Kraemer, Melanie, Gerlach, Frank, Schuh, Reinhard, Burmester, Thorsten, Hankeln, Thomas

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Factor VIII-eGFP fusion proteins with preserved functional activity for the analysis of the early secretory pathway of factor VIII von Heinz, Stefan, Schüttrumpf, Jörg, Simpson, Jeremy C., Pepperkok, Rainer, Nicolaes, Gerry A., Abriss, Daniela, Milanov, Peter, Roth, Stefanie, 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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Combined +58 and +55 BCL11A enhancer Editing Yields Exceptional Efficiency, Specificity and HbF Induction in Human and NHP Preclinical Models von Zeng, Jing, Demirci, Selami, Nguyen, My Anh, Lin, Linda Yingqi, Maitland, Stacy A., Mintzer, Esther, Wu, Yuxuan, Pellin, Danilo, Tangprasittipap, Amornrat, Vong, Chokdee, Porter, Shaina N., Luk, Kevin, Liu, Pengpeng, Katta, Varun, Ciuculescu, Marioara-Felicia, Abriss, Daniela, Hsu, Jonathan, Uchida, Naoya, Essawi, Khaled, Donahue, Robert, Petri, Karl, Pattanayak, Vikram, Pinello, Luca, Brendel, Christian, Williams, David A, Manis, John P, Tsai, Shengdar Q., Pruett-Miller, Shondra M., Joung, J. Keith, Songdej, Duantida, Hongeng, Suradej, Armant, Myriam, Wolfe, Scot A., Tisdale, John F., Bauer, Daniel E.

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Flipping the Switch: Initial Results of Genetic Targeting of the Fetal to Adult Globin Switch in Sickle Cell Patients von Esrick, Erica B., Brendel, Christian, Manis, John P, Armant, Myriam A, Negre, Helene, Dansereau, Colleen, Ciuculescu, Marioara Felicia, Patriarca, Stephanie, Mackinnon, Brenda, Daley, Heather, Richard, Shanna, Abriss, Daniela, Maxwell, Renee, Braunewell, Stephen, Christiansen, Lauryn, Nikiforow, Sarah, Ritz, Jerome, Achebe, Maureen, Negre, Olivier, Lehmann, Leslie E., Heeney, Matthew M, Biffi, Alessandra, Williams, David A.

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CD133-targeted Gene Transfer Into Long-term Repopulating Hematopoietic Stem Cells von Brendel, Christian, Goebel, Benjamin, Daniela, Abriss, Brugman, Martijn, Kneissl, Sabrina, Schwäble, Joachim, Kaufmann, Kerstin B, Müller-Kuller, Uta, Kunkel, Hana, Chen-Wichmann, Linping, Abel, Tobias, Serve, Hubert, Bystrykh, Leonid, Buchholz, Christian J, Grez, Manuel

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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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Engineered factor IX variants bypass FVIII and correct hemophilia A phenotype in mice von Milanov, Peter, Ivanciu, Lacramioara, Abriss, Daniela, Quade-Lyssy, Patricia, Miesbach, Wolfgang, Alesci, Sonja, Tonn, Torsten, Grez, Manuel, Seifried, Erhard, Schüttrumpf, Jörg

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Improving Hematopoietic Stem Cell Based Gene Therapy By Targeting CD133+ Cells von Goebel, Benjamin, Brendel, Christian, Abriss, Daniela, Kneissl, Sabrina, Brugman, Martijn, Buchholz, Christian J., Serve, Hubert, Grez, Manuel

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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, Grez, Manuel

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FactorVIII-eGFP fusion proteins with preserved functional activity for the analysis of the early secretory pathway of factor VIII von HEINZ, Stefan, SCHÜTTRUMPF, Jörg, SIMPSON, Jeremy C, PEPPERKOK, Rainer, NICOLAES, Gerry A, ABRISS, Daniela, MILANOV, Peter, ROTH, Stefanie, SEIFRIED, Erhard, TONN, Torsten

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

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