Search Results - "Wiebking, Volker"

Gene correction for SCID-X1 in long-term hematopoietic stem cells by Pavel-Dinu, Mara, Wiebking, Volker, Dejene, Beruh T., Srifa, Waracharee, Mantri, Sruthi, Nicolas, Carmencita E., Lee, Ciaran, Bao, Gang, Kildebeck, Eric J., Punjya, Niraj, Sindhu, Camille, Inlay, Matthew A., Saxena, Nivedita, DeRavin, Suk See, Malech, Harry, Roncarolo, Maria Grazia, Weinberg, Kenneth I., Porteus, Matthew H.

“…Gene correction in human long-term hematopoietic stem cells (LT-HSCs) could be an effective therapy for monogenic diseases of the blood and immune system. Here…”
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A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells by Vakulskas, Christopher A., Dever, Daniel P., Rettig, Garrett R., Turk, Rolf, Jacobi, Ashley M., Collingwood, Michael A., Bode, Nicole M., McNeill, Matthew S., Yan, Shuqi, Camarena, Joab, Lee, Ciaran M., Park, So Hyun, Wiebking, Volker, Bak, Rasmus O., Gomez-Ospina, Natalia, Pavel-Dinu, Mara, Sun, Wenchao, Bao, Gang, Porteus, Matthew H., Behlke, Mark A.

“…Translation of the CRISPR–Cas9 system to human therapeutics holds high promise. However, specificity remains a concern especially when modifying stem cell…”
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Metabolic engineering generates a transgene-free safety switch for cell therapy by Wiebking, Volker, Patterson, James O., Martin, Renata, Chanda, Monica K., Lee, Ciaran M., Srifa, Waracharee, Bao, Gang, Porteus, Matthew H.

“…Safeguard mechanisms can ameliorate the potential risks associated with cell therapies but currently rely on the introduction of transgenes. This limits their…”
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Highly Efficient and Marker-free Genome Editing of Human Pluripotent Stem Cells by CRISPR-Cas9 RNP and AAV6 Donor-Mediated Homologous Recombination by Martin, Renata M., Ikeda, Kazuya, Cromer, M. Kyle, Uchida, Nobuko, Nishimura, Toshinobu, Romano, Rosa, Tong, Andrew J., Lemgart, Viktor T., Camarena, Joab, Pavel-Dinu, Mara, Sindhu, Camille, Wiebking, Volker, Vaidyanathan, Sriram, Dever, Daniel P., Bak, Rasmus O., Laustsen, Anders, Lesch, Benjamin J., Jakobsen, Martin R., Sebastiano, Vittorio, Nakauchi, Hiromitsu, Porteus, Matthew H.

“…Genome editing of human pluripotent stem cells (hPSCs) provides powerful opportunities for in vitro disease modeling, drug discovery, and personalized stem…”
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T-cell replete haploidentical bone marrow transplantation and post-transplant cyclophosphamide for patients with inborn errors by Kurzay, Mathias, Hauck, Fabian, Schmid, Irene, Wiebking, Volker, Eichinger, Anna, Jung, Eva, Boekstegers, Ann, Feuchtinger, Tobias, Klein, Christoph, Albert, Michael H

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Maturation of Platelet Function During Murine Fetal Development In Vivo by Margraf, Andreas, Nussbaum, Claudia, Rohwedder, Ina, Klapproth, Sarah, Kurz, Angela R M, Florian, Annamaria, Wiebking, Volker, Pircher, Joachim, Pruenster, Monika, Immler, Roland, Dietzel, Steffen, Kremer, Ludmila, Kiefer, Friedemann, Moser, Markus, Flemmer, Andreas W, Quackenbush, Elizabeth, von Andrian, Ulrich H, Sperandio, Markus

“…Platelet function has been intensively studied in the adult organism. However, little is known about the function and hemostatic capacity of platelets in the…”
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Genome editing of donor-derived T-cells to generate allogenic chimeric antigen receptor-modified T cells: Optimizing αβ T cell-depleted haploidentical hematopoietic stem cell transplantation by Wiebking, Volker, Lee, Ciaran M, Mostrel, Nathalie, Lahiri, Premanjali, Bak, Rasmus, Bao, Gang, Roncarolo, Maria Grazia, Bertaina, Alice, Porteus, Matthew H

“…Allogeneic hematopoietic stem cell transplantation is an effective therapy for high-risk leukemias. In children, graft manipulation based on the selective…”
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Author Correction: Gene correction for SCID-X1 in long-term hematopoietic stem cells by Pavel-Dinu, Mara, Wiebking, Volker, Dejene, Beruh T., Srifa, Waracharee, Mantri, Sruthi, Nicolas, Carmencita E., Lee, Ciaran, Bao, Gang, Kildebeck, Eric J., Punjya, Niraj, Sindhu, Camille, Inlay, Matthew A., Saxena, Nivedita, DeRavin, Suk See, Malech, Harry, Roncarolo, Maria Grazia, Weinberg, Kenneth I., Porteus, Matthew H.

“…The original version of this Article omitted the following from the Acknowledgements: “G.B. acknowledges the support from the Cancer Prevention and Research…”
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Author Correction: Gene correction for SCID-X1 in long-term hematopoietic stem cells by Pavel-Dinu, Mara, Wiebking, Volker, Dejene, Beruh T., Srifa, Waracharee, Mantri, Sruthi, Nicolas, Carmencita E., Lee, Ciaran, Bao, Gang, Kildebeck, Eric J., Punjya, Niraj, Sindhu, Camille, Inlay, Matthew A., Saxena, Nivedita, DeRavin, Suk See, Malech, Harry, Roncarolo, Maria Grazia, Weinberg, Kenneth I., Porteus, Matthew H.

“…An amendment to this paper has been published and can be accessed via a link at the top of the paper…”
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Impairment of endothelial progenitor cell function and vascularization capacity by aldosterone in mice and humans by THUM, Thomas, SCHMITTER, Kerstin, FLEISSNER, Felix, WIEBKING, Volker, DIETRICH, Bernd, WIDDER, Julian D, JAZBUTYTE, Virginija, HAHNER, Stefanie, ERTL, Georg, BAUERSACHS, Johann

“…Hyperaldosteronism is associated with vascular injury and increased cardiovascular events. Bone marrow-derived endothelial progenitor cells (EPCs) play an…”
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Abstract 15594: Decreasing the High Risk of Ponatinib-Induced Cardiovascular Toxicity in Chronic Myeloid Leukemia by Hnatiuk Hnatiuk, Anna P, Bruyneel, Arne A, Taylor, Dhanir, Pandrala, Mallesh, Dheeraj, Arpit, Li, Wenqi, Serrano, Ricardo, Feyen, Dries, Vu, Michelle, Amatya, Prashila, Saloni, Gupta, Nakauchi, Yusuke, Morgado, Isabel, Wiebking, Volker, Liao, Ronglih, Porteus, Matthew, Majeti, Ravindra, Malhotra, Sanjay, Mercola, Mark

“…IntroductionCardiovascular (CV) toxicity of cancer therapeutics is a growing problem. Ponatinib is one of the most cardiotoxic Tyrosine Kinase Inhibitors…”
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Reduced toxicity, myeloablative HLA-haploidentical hematopoietic stem cell transplantation with post-transplantation cyclophosphamide for sickle cell disease by Wiebking, Volker, Hütker, Sebastian, Schmid, Irene, Immler, Stefanie, Feuchtinger, Tobias, Albert, Michael H.

“…Allogeneic hematopoietic stem cell transplantation (HSCT) offers the possibility of cure for sickle cell disease (SCD) patients. Unfortunately, the probability…”
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Outcome of HSCT in Adolescents and Young Adults with Non-SCID Primary Immunodeficiencies by Albert, Michael H., MD, Wiebking, Volker, MD, Notheis, Gundula, MD, Hauck, Fabian, MD, Aumann, Volker, MD, Koletzko, Sibylle, Belohradsky, Bernd H., MD, Führer, Monika, MD, Renner, Ellen D., MD, Tischer, Johanna, MD, Klein, Christoph, MD, Schmid, Irene, MD

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Reengineering Ponatinib to Minimize Cardiovascular Toxicity by Hnatiuk, Anna P, Bruyneel, Arne A N, Tailor, Dhanir, Pandrala, Mallesh, Dheeraj, Arpit, Li, Wenqi, Serrano, Ricardo, Feyen, Dries A M, Vu, Michelle M, Amatya, Prashila, Gupta, Saloni, Nakauchi, Yusuke, Morgado, Isabel, Wiebking, Volker, Liao, Ronglih, Porteus, Matthew H, Majeti, Ravindra, Malhotra, Sanjay V, Mercola, Mark

“…Small molecule tyrosine kinase inhibitors (TKI) have revolutionized cancer treatment and greatly improved patient survival. However, life-threatening…”
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Subcutaneous Mosunetuzumab Is Active with a Manageable Safety Profile in Patients (pts) with Relapsed/Refractory (R/R) B-Cell Non-Hodgkin Lymphomas (B-NHLs): Updated Results from a Phase I/II Study by Budde, Elizabeth L., Bartlett, Nancy L., Giri, Pratyush, Schuster, Stephen J., Assouline, Sarit, Yoon, Sung-Soo, Fay, Keith, Matasar, Matthew J., Gutierrez, Norma C., Marlton, Paula, Dreyling, Martin, Yoon, Dok Hyun, Hess, Georg, Radford, John, Wiebking, Volker, Yin, Shen, Cybulski, Eva, Turner, David C., Huang, Huang, Zhou, Mingzhu, Penuel, Elicia, Wei, Michael C., Sehn, Laurie H.

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IPEX due to an exon 7 skipping FOXP3 mutation with autoimmune diabetes mellitus cured by selective T Reg cell engraftment by Magg, Thomas, Wiebking, Volker, Conca, Raffaele, Krebs, Stefan, Arens, Stefan, Schmid, Irene, Klein, Christoph, Albert, Michael H, Hauck, Fabian

“…Immune dysregulation, polyendocrinopathy, enteropathy, X-linked (IPEX) syndrome is a rare inherited disorder leading to severe organ-specific autoimmunity…”
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Genome editing of donor-derived T cells to generate allogeneic chimeric antigen receptor-modified T cells: optimizing αβ T-cell-depleted haploidentical hematopoietic stem cell transplantation by Wiebking, Volker, Lee, Ciaran M., Mostrel, Nathalie, Lahiri, Premanjali, Bak, Rasmus, Bao, Gang, Roncarolo, Maria Grazia, Bertaina, Alice, Porteus, Matthew H.

“…Allogeneic hematopoietic stem cell transplantation is an effective therapy for high-risk leukemias. In children, graft manipulation based on the selective…”
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Subcutaneous (SC) Administration of Mosunetuzumab with Cycle 1 Step-up Dosing Is Tolerable and Active in Patients with Relapsed/Refractory B-Cell Non-Hodgkin Lymphomas (R/R B-NHL): Initial Results from a Phase I/II Study by Bartlett, Nancy L., Giri, Pratyush, Budde, L. Elizabeth, Schuster, Stephen J., Assouline, Sarit, Matasar, Matthew J., Yoon, Sung-Soo, Canales, Miguel, Gutierrez, Norma C., Fay, Keith, Cheah, Chan-Yoon, Marlton, Paula, Wiebking, Volker, Yin, Shen, To, Iris, Li, Chi-Chung, Huang, Huang, Zhou, Mingzhu, Penuel, Elicia, O'Hear, Carol, Sehn, Laurie H.

“…Background: Cytokine release syndrome (CRS) is a potentially serious complication of T-cell engaging immunotherapy. Effective measures are needed to reduce the…”
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IPEX due to an exon 7 skipping FOXP3 mutation with autoimmune diabetes mellitus cured by selective TReg cell engraftment by Magg, Thomas, Wiebking, Volker, Conca, Raffaele, Krebs, Stefan, Arens, Stefan, Schmid, Irene, Klein, Christoph, Albert, Michael H., Hauck, Fabian

“…Immune dysregulation, polyendocrinopathy, enteropathy, X-linked (IPEX) syndrome is a rare inherited disorder leading to severe organ-specific autoimmunity…”
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Organic nitrates differentially modulate circulating endothelial progenitor cells and endothelial function in patients with symptomatic coronary artery disease by Thum, Thomas, Wiebking, Volker, Ertl, Georg, Bauersachs, Johann

“…Symptomatic coronary artery disease (CAD) is usually treated with organic nitrates. Endothelial progenitor cells (EPCs) are a circulating cell population…”
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