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.

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Progress Toward In Vivo Use of siRNAs-II von Rettig, Garrett R, Behlke, Mark A

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Optimized design parameters for CRISPR Cas9 and Cas12a homology-directed repair von Schubert, Mollie S., Thommandru, Bernice, Woodley, Jessica, Turk, Rolf, Yan, Shuqi, Kurgan, Gavin, McNeill, Matthew S., Rettig, Garrett R.

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Simplified CRISPR tools for efficient genome editing and streamlined protocols for their delivery into mammalian cells and mouse zygotes von Jacobi, Ashley M., Rettig, Garrett R., Turk, Rolf, Collingwood, Michael A., Zeiner, Sarah A., Quadros, Rolen M., Harms, Donald W., Bonthuis, Paul J., Gregg, Christopher, Ohtsuka, Masato, Gurumurthy, Channabasavaiah B., Behlke, Mark A.

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CRISPECTOR provides accurate estimation of genome editing translocation and off-target activity from comparative NGS data von Amit, Ido, Iancu, Ortal, Levy-Jurgenson, Alona, Kurgan, Gavin, McNeill, Matthew S., Rettig, Garrett R., Allen, Daniel, Breier, Dor, Ben Haim, Nimrod, Wang, Yu, Anavy, Leon, Hendel, Ayal, Yakhini, Zohar

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Shuttle peptide delivers base editor RNPs to rhesus monkey airway epithelial cells in vivo von Kulhankova, Katarina, Traore, Soumba, Cheng, Xue, Benk-Fortin, Hadrien, Hallée, Stéphanie, Harvey, Mario, Roberge, Joannie, Couture, Frédéric, Kohli, Sajeev, Gross, Thomas J., Meyerholz, David K., Rettig, Garrett R., Thommandru, Bernice, Kurgan, Gavin, Wohlford-Lenane, Christine, Hartigan-O’Connor, Dennis J., Yates, Bradley P., Newby, Gregory A., Liu, David R., Tarantal, Alice F., Guay, David, McCray, Paul B.

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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.

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Increasing CRISPR Efficiency and Measuring Its Specificity in HSPCs Using a Clinically Relevant System von Shapiro, Jenny, Iancu, Ortal, Jacobi, Ashley M., McNeill, Matthew S., Turk, Rolf, Rettig, Garrett R., Amit, Ido, Tovin-Recht, Adi, Yakhini, Zohar, Behlke, Mark A., Hendel, Ayal

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Comparative analysis of CRISPR off-target discovery tools following ex vivo editing of CD34+ hematopoietic stem and progenitor cells von Cromer, M. Kyle, Majeti, Kiran R., Rettig, Garrett R., Murugan, Karthik, Kurgan, Gavin L., Bode, Nicole M., Hampton, Jessica P., Vakulskas, Christopher A., Behlke, Mark A., Porteus, Matthew H.

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Targeting a cytokine checkpoint enhances the fitness of armored cord blood CAR-NK cells von Daher, May, Basar, Rafet, Gokdemir, Elif, Baran, Natalia, Uprety, Nadima, Nunez Cortes, Ana Karen, Mendt, Mayela, Kerbauy, Lucila Nassif, Banerjee, Pinaki P., Shanley, Mayra, Imahashi, Nobuhiko, Li, Li, Lim, Francesca Lorraine Wei Inng, Fathi, Mohsen, Rezvan, Ali, Mohanty, Vakul, Shen, Yifei, Shaim, Hila, Lu, Junjun, Ozcan, Gonca, Ensley, Emily, Kaplan, Mecit, Nandivada, Vandana, Bdiwi, Mustafa, Acharya, Sunil, Xi, Yuanxin, Wan, Xinhai, Mak, Duncan, Liu, Enli, Jiang, Xin Ru, Ang, Sonny, Muniz-Feliciano, Luis, Li, Ye, Wang, Jing, Kordasti, Shahram, Petrov, Nedyalko, Varadarajan, Navin, Marin, David, Brunetti, Lorenzo, Skinner, Richard J., Lyu, Shangrong, Silva, Leiser, Turk, Rolf, Schubert, Mollie S., Rettig, Garrett R., McNeill, Matthew S., Kurgan, Gavin, Behlke, Mark A., Li, Heng, Fowlkes, Natalie W., Chen, Ken, Konopleva, Marina, Champlin, Richard E., Shpall, Elizabeth J., Rezvani, Katayoun

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CRISPAltRations: A validated cloud-based approach for interrogation of double-strand break repair mediated by CRISPR genome editing von Kurgan, Gavin, Turk, Rolf, Li, Heng, Roberts, Nathan, Rettig, Garrett R., Jacobi, Ashley M., Tso, Lauren, Sturgeon, Morgan, Mertens, Massimo, Noten, Roel, Florus, Kurt, Behlke, Mark A., Wang, Yu, McNeill, Matthew S.

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Potency of siRNA versus shRNA mediated knockdown in vivo von McAnuff, Marie A., Rettig, Garrett R., Rice, Kevin G.

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Knockdown of β-catenin with Dicer-Substrate siRNAs Reduces Liver Tumor Burden In vivo von Dudek, Henryk, Wong, Darren H, Arvan, Rokhand, Shah, Anee, Wortham, Kathleen, Ying, Bo, Diwanji, Rohan, Zhou, Wei, Holmes, Benjamin, Yang, Hailin, Cyr, Wendy A, Zhou, Yi, Shah, Aalok, Farkiwala, Ruchir, Lee, Michael, Li, Yiting, Rettig, Garrett R, Collingwood, Michael A, Basu, Sujit K, Behlke, Mark A, Brown, Bob D

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High-level correction of the sickle mutation is amplified in vivo during erythroid differentiation von Magis, Wendy, DeWitt, Mark A., Wyman, Stacia K., Vu, Jonathan T., Heo, Seok-Jin, Shao, Shirley J., Hennig, Finn, Romero, Zulema G., Campo-Fernandez, Beatriz, Said, Suzanne, McNeill, Matthew S., Rettig, Garrett R., Sun, Yongming, Wang, Yu, Behlke, Mark A., Kohn, Donald B., Boffelli, Dario, Walters, Mark C., Corn, Jacob E., Martin, David I.K.

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Large-scale GMP-compliant CRISPR-Cas9–mediated deletion of the glucocorticoid receptor in multivirus-specific T cells von Basar, Rafet, Daher, May, Uprety, Nadima, Gokdemir, Elif, Alsuliman, Abdullah, Ensley, Emily, Ozcan, Gonca, Mendt, Mayela, Hernandez Sanabria, Mayra, Kerbauy, Lucila Nassif, Nunez Cortes, Ana Karen, Li, Li, Banerjee, Pinaki P., Muniz-Feliciano, Luis, Acharya, Sunil, Fowlkes, Natalie W., Lu, Junjun, Li, Sufang, Mielke, Stephan, Kaplan, Mecit, Nandivada, Vandana, Bdaiwi, Mustafa, Kontoyiannis, Alexander D., Li, Ye, Liu, Enli, Ang, Sonny, Marin, David, Brunetti, Lorenzo, Gundry, Michael C., Turk, Rolf, Schubert, Mollie S., Rettig, Garrett R., McNeill, Matthew S., Kurgan, Gavin, Behlke, Mark A., Champlin, Richard, Shpall, Elizabeth J., Rezvani, Katayoun

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Non-viral gene delivery: from the needle to the nucleus von Rettig, Garrett R, Rice, Kevin G

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Quantitative bioluminescence imaging of transgene expression in vivo von Rettig, Garrett R., McAnuff, Marie, Liu, Dijie, Kim, Ji-Seon, Rice, Kevin G.

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Synthetic PEGylated Glycoproteins and Their Utility in Gene Delivery von Chen, Chang-po, Kim, Ji-seon, Liu, Dijie, Rettig, Garrett R, McAnuff, Marie A, Martin, Molly E, Rice, Kevin G

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Quantitative In Vivo Imaging of Non-viral-Mediated Gene Expression and RNAi-Mediated Knockdown von Rettig, Garrett R., Rice, Kevin G.

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An “off-the-shelf” CD2 universal CAR-T therapy for T-cell malignancies von Xiang, Jingyu, Devenport, Jessica M., Carter, Alun J., Staser, Karl W., Kim, Miriam Y., O’ Neal, Julie, Ritchey, Julie K., Rettig, Michael P., Gao, Feng, Rettig, Garrett, Turk, Rolf, Lee, Byung Ha, Cooper, Matthew L., DiPersio, John F.

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