Identification of preexisting adaptive immunity to Cas9 proteins in humans von Charlesworth, Carsten T., Deshpande, Priyanka S., Dever, Daniel P., Camarena, Joab, Lemgart, Viktor T., Cromer, M. Kyle, Vakulskas, Christopher A., Collingwood, Michael A., Zhang, Liyang, Bode, Nicole M., Behlke, Mark A., Dejene, Beruh, Cieniewicz, Brandon, Romano, Rosa, Lesch, Benjamin J., Gomez-Ospina, Natalia, Mantri, Sruthi, Pavel-Dinu, Mara, Weinberg, Kenneth I., Porteus, Matthew H.

Volltext

A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells von 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.

Volltext

Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice von Wilkinson, Adam C., Dever, Daniel P., Baik, Ron, Camarena, Joab, Hsu, Ian, Charlesworth, Carsten T., Morita, Chika, Nakauchi, Hiromitsu, Porteus, Matthew H.

Volltext

CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells von Dever, Daniel P., Bak, Rasmus O., Reinisch, Andreas, Camarena, Joab, Washington, Gabriel, Nicolas, Carmencita E., Pavel-Dinu, Mara, Saxena, Nivi, Wilkens, Alec B., Mantri, Sruthi, Uchida, Nobuko, Hendel, Ayal, Narla, Anupama, Majeti, Ravindra, Weinberg, Kenneth I., Porteus, Matthew H.

Volltext

The changing landscape of gene editing in hematopoietic stem cells: a step towards Cas9 clinical translation von Dever, Daniel P, Porteus, Matthew H

Volltext

Highly Efficient and Marker-free Genome Editing of Human Pluripotent Stem Cells by CRISPR-Cas9 RNP and AAV6 Donor-Mediated Homologous Recombination von 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.

Volltext

Global Transcriptional Response to CRISPR/Cas9-AAV6-Based Genome Editing in CD34+ Hematopoietic Stem and Progenitor Cells von Cromer, M. Kyle, Vaidyanathan, Sriram, Ryan, Daniel E., Curry, Bo, Lucas, Anne Bergstrom, Camarena, Joab, Kaushik, Milan, Hay, Sarah R., Martin, Renata M., Steinfeld, Israel, Bak, Rasmus O., Dever, Daniel P., Hendel, Ayal, Bruhn, Laurakay, Porteus, Matthew H.

Volltext

Highly efficient editing of the β-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease von Park, So Hyun, Lee, Ciaran M, Dever, Daniel P, Davis, Timothy H, Camarena, Joab, Srifa, Waracharee, Zhang, Yankai, Paikari, Alireza, Chang, Alicia K, Porteus, Matthew H, Sheehan, Vivien A, Bao, Gang

Volltext

Multiplexed genetic engineering of human hematopoietic stem and progenitor cells using CRISPR/Cas9 and AAV6 von Bak, Rasmus O, Dever, Daniel P, Reinisch, Andreas, Cruz Hernandez, David, Majeti, Ravindra, Porteus, Matthew H

Volltext

The TRACE-Seq method tracks recombination alleles and identifies clonal reconstitution dynamics of gene targeted human hematopoietic stem cells von Sharma, Rajiv, Dever, Daniel P., Lee, Ciaran M., Azizi, Armon, Pan, Yidan, Camarena, Joab, Köhnke, Thomas, Bao, Gang, Porteus, Matthew H., Majeti, Ravindra

Volltext

Priming Human Repopulating Hematopoietic Stem and Progenitor Cells for Cas9/sgRNA Gene Targeting von Charlesworth, Carsten T., Camarena, Joab, Cromer, M. Kyle, Vaidyanathan, Sriram, Bak, Rasmus O., Carte, Jason M., Potter, Jason, Dever, Daniel P., Porteus, Matthew H.

Volltext

CRISPR/Cas9 genome editing in human hematopoietic stem cells von Bak, Rasmus O, Dever, Daniel P, Porteus, Matthew H

Volltext

Transient inhibition of 53BP1 increases the frequency of targeted integration in human hematopoietic stem and progenitor cells von Baik, Ron, Cromer, M. Kyle, Glenn, Steve E., Vakulskas, Christopher A., Chmielewski, Kay O., Dudek, Amanda M., Feist, William N., Klermund, Julia, Shipp, Suzette, Cathomen, Toni, Dever, Daniel P., Porteus, Matthew H.

Volltext

Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells von Cromer, M. Kyle, Camarena, Joab, Martin, Renata M., Lesch, Benjamin J., Vakulskas, Christopher A., Bode, Nicole M., Kurgan, Gavin, Collingwood, Michael A., Rettig, Garrett R., Behlke, Mark A., Lemgart, Viktor T., Zhang, Yankai, Goyal, Ankush, Zhao, Feifei, Ponce, Ezequiel, Srifa, Waracharee, Bak, Rasmus O., Uchida, Naoya, Majeti, Ravindra, Sheehan, Vivien A., Tisdale, John F., Dever, Daniel P., Porteus, Matthew H.

Volltext

Functional screening in human HSPCs identifies optimized protein-based enhancers of Homology Directed Repair von Perez-Bermejo, Juan A., Efagene, Oghene, Matern, William M., Holden, Jeffrey K., Kabir, Shaheen, Chew, Glen M., Andreoletti, Gaia, Catton, Eniola, Ennis, Craig L., Garcia, Angelica, Gerstenberg, Trevor L., Hill, Kaisle A., Jain, Aayami, Krassovsky, Kristina, Lalisan, Cassandra D., Lord, Daniel, Quejarro, B. Joy, Sales-Lee, Jade, Shah, Meet, Silva, Brian J., Skowronski, Jason, Strukov, Yuri G., Thomas, Joshua, Veraz, Michael, Vijay, Twaritha, Wallace, Kirby A., Yuan, Yue, Grogan, Jane L., Wienert, Beeke, Lahiri, Premanjali, Treusch, Sebastian, Dever, Daniel P., Soros, Vanessa B., Partridge, James R., Seim, Kristen L.

Volltext

Aryl hydrocarbon receptor deletion in cerebellar granule neuron precursors impairs neurogenesis von Dever, Daniel P., Adham, Zachariah O., Thompson, Bryan, Genestine, Matthieu, Cherry, Jonathan, Olschowka, John A., DiCicco‐Bloom, Emanuel, Opanashuk, Lisa A.

Volltext

The Aryl Hydrocarbon Receptor Contributes to the Proliferation of Human Medulloblastoma Cells von Dever, Daniel P., Opanashuk, Lisa A.

Volltext

Technical considerations for the use of CRISPR/Cas9 in hematology research von Gundry, Michael C., Dever, Daniel P., Yudovich, David, Bauer, Daniel E., Haas, Simon, Wilkinson, Adam C., Singbrant, Sofie

Volltext

Clinically relevant gene editing in hematopoietic stem cells for the treatment of pyruvate kinase deficiency von Fañanas-Baquero, Sara, Quintana-Bustamante, Oscar, Dever, Daniel P., Alberquilla, Omaira, Sanchez-Dominguez, Rebeca, Camarena, Joab, Ojeda-Perez, Isabel, Dessy-Rodriguez, Mercedes, Turk, Rolf, Schubert, Mollie S., Lattanzi, Annalisa, Xu, Liwen, Lopez-Lorenzo, Jose L., Bianchi, Paola, Bueren, Juan A., Behlke, Mark A., Porteus, Matthew, Segovia, Jose-Carlos

Volltext

CRISPR/Cas9 Genome Engineering in Engraftable Human Brain-Derived Neural Stem Cells von Dever, Daniel P., Scharenberg, Samantha G., Camarena, Joab, Kildebeck, Eric J., Clark, Joseph T., Martin, Renata M., Bak, Rasmus O., Tang, Yuming, Dohse, Monika, Birgmeier, Johannes A., Jagadeesh, Karthik A., Bejerano, Gill, Tsukamoto, Ann, Gomez-Ospina, Natalia, Uchida, Nobuko, Porteus, Matthew H.

Volltext