Unconstrained genome targeting with near-PAMless engineered CRISPR-Cas9 variants von Walton, Russell T, Christie, Kathleen A, Whittaker, Madelynn N, Kleinstiver, Benjamin P

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(56) A Preclinical Study of CRISPR Porcine Cell Gene Editing Combined with IL-10 Gene Delivery for Donor Lung Immunomodulation von Yoshiyasu, N., Mesaki, K., Guan, Z., Juvet, S., Kleinstiver, B., Cypel, M., Liu, M., Keshavjee, S.

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Engineered CRISPR-Cas9 nucleases with altered PAM specificities von Kleinstiver, Benjamin P., Prew, Michelle S., Tsai, Shengdar Q., Topkar, Ved V., Nguyen, Nhu T., Zheng, Zongli, Gonzales, Andrew P. W., Li, Zhuyun, Peterson, Randall T., Yeh, Jing-Ruey Joanna, Aryee, Martin J., Joung, J. Keith

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(55) Genetic Engineering for Combined Early and Long-Term Immunomodulation in the Donor Lung after Transplantation von Mesaki, K., Juvet, S., Yeung, J., Mangat, H., Dickie, C., Guan, Z., Shathasivam, P., Hu, J., Davidson, A., Kleinstiver, B., Cypel, M., Liu, M., Keshavjee, S.

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(8) - CRISPR-Cas Genome Editing in Ex Vivo Human Lungs von Mesaki, K., Yamamoto, H., Juvet, S., Yeung, J., Guan, Z., Yao, Y., Chen, M., Gokhale, H., Shan, H., Wang, A., Wilson, G., Mariscal, A., Hu, J., Davidson, A., Kleinstiver, B., Cypel, M., Liu, M., Keshavjee, S.

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Derepression of the IL-10 Gene by CRISPR-Cas Genome Editing as a Therapeutic Strategy for Persistent Immunomodulation of Donor Lungs for Transplantation von Mesaki, K., Juvet, S., Yeung, J., Ivakine, E., Guan, Z., Shathasivam, P., Akhter, A., Wilson, G., Kleinstiver, B., Hu, J., Davidson, A., Cypel, M., Liu, M., Keshavjee, S.

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Strand-specific Contacts and Divalent Metal Ion Regulate Double-strand Break Formation by the GIY-YIG Homing Endonuclease I-BmoI von Carter, Jonathan M., Friedrich, Nancy C., Kleinstiver, Benjamin, Edgell, David R.

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Homosnomobilius von Damschroder, Allen D., Kleinstiver, Benjamin J.

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Enhanced proofreading governs CRISPR–Cas9 targeting accuracy von Chen, Janice S., Dagdas, Yavuz S., Kleinstiver, Benjamin P., Welch, Moira M., Sousa, Alexander A., Harrington, Lucas B., Sternberg, Samuel H., Joung, J. Keith, Yildiz, Ahmet, Doudna, Jennifer A.

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Treatment of monogenic and digenic dominant genetic hearing loss by CRISPR-Cas9 ribonucleoprotein delivery in vivo von Tao, Yong, Lamas, Veronica, Du, Wan, Zhu, Wenliang, Li, Yiran, Whittaker, Madelynn N., Zuris, John A., Thompson, David B., Rameshbabu, Arun Prabhu, Shu, Yilai, Gao, Xue, Hu, Johnny H., Pei, Charles, Kong, Wei-Jia, Liu, Xuezhong, Wu, Hao, Kleinstiver, Benjamin P., Liu, David R., Chen, Zheng-Yi

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Homosnomobilius von Damschroder, AD, Kleinstiver, B J

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In vivo engineering of lymphocytes after systemic exosome-associated AAV delivery von Breuer, Cort B., Hanlon, Killian S., Natasan, Jeya-shree, Volak, Adrienn, Meliani, Amine, Mingozzi, Federico, Kleinstiver, Benjamin P., Moon, James J., Maguire, Casey A.

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Enhanced homology-directed repair for highly efficient gene editing in hematopoietic stem/progenitor cells von De Ravin, Suk See, Brault, Julie, Meis, Ronald J., Liu, Siyuan, Li, Linhong, Pavel-Dinu, Mara, Lazzarotto, Cicera R., Liu, Taylor, Koontz, Sherry M., Choi, Uimook, Sweeney, Colin L., Theobald, Narda, Lee, GaHyun, Clark, Aaron B., Burkett, Sandra S., Kleinstiver, Benjamin P., Porteus, Matthew H., Tsai, Shengdar, Kuhns, Douglas B., Dahl, Gary A., Headey, Stephen, Wu, Xiaolin, Malech, Harry L.

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CRISPR targeting of mmu-miR-21a through a single adeno-associated virus vector prolongs survival of glioblastoma-bearing mice von Nieland, Lisa, Vrijmoet, Anne B., Jetten, Isabelle W., Rufino-Ramos, David, de Reus, Alexandra J.E.M., Breyne, Koen, Kleinstiver, Benjamin P., Maguire, Casey A., Broekman, Marike L.D., Breakefield, Xandra O., Abels, Erik R.

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CRISPR-targeted MAGT1 insertion restores XMEN patient hematopoietic stem cells and lymphocytes von Brault, Julie, Liu, Taylor, Bello, Ezekiel, Liu, Siyuan, Sweeney, Colin L., Meis, Ronald J., Koontz, Sherry, Corsino, Cristina, Choi, Uimook, Vayssiere, Guillaume, Bosticardo, Marita, Dowdell, Kennichi, Lazzarotto, Cicera R., Clark, Aaron B., Notarangelo, Luigi D., Ravell, Juan C., Lenardo, Michael J., Kleinstiver, Benjamin P., Tsai, Shengdar Q., Wu, Xiaolin, Dahl, Gary A., Malech, Harry L., De Ravin, Suk See

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Isocitrate Dehydrogenase Mutations Confer Dasatinib Hypersensitivity and SRC Dependence in Intrahepatic Cholangiocarcinoma von Saha, Supriya K, Gordan, John D, Kleinstiver, Benjamin P, Vu, Phuong, Najem, Mortada S, Yeo, Jia-Chi, Shi, Lei, Kato, Yasutaka, Levin, Rebecca S, Webber, James T, Damon, Leah J, Egan, Regina K, Greninger, Patricia, McDermott, Ultan, Garnett, Mathew J, Jenkins, Roger L, Rieger-Christ, Kimberly M, Sullivan, Travis B, Hezel, Aram F, Liss, Andrew S, Mizukami, Yusuke, Goyal, Lipika, Ferrone, Cristina R, Zhu, Andrew X, Joung, J Keith, Shokat, Kevan M, Benes, Cyril H, Bardeesy, Nabeel

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Cell-based artificial APC resistant to lentiviral transduction for efficient generation of CAR-T cells from various cell sources von Schmidts, Andrea, Marsh, Leah C, Srivastava, Ambike A, Bouffard, Amanda A, Boroughs, Angela C, Scarfò, Irene, Larson, Rebecca C, Bedoya, Felipe, Choi, Bryan D, Frigault, Matthew J, Bailey, Stefanie R, Leick, Mark B, Vatsa, Sonika, Kann, Michael C, Prew, Michelle S, Kleinstiver, Benjamin P, Joung, J Keith, Maus, Marcela V

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Enhanced Proofreading Governs CRISPR-Cas9 Targeting Accuracy von Chen, Janice S., Dagdas, Yavuz S., Kleinstiver, Benjamin P., Welch, Moira M., Sousa, Alexander A., Harrington, Lucas B., Sternberg, Samuel H., Joung, Keith J., Yildiz, Ahmet, Doudna, Jennifer A.

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Highly Efficient & Specific Repair of MAGT1 Mutation in Xmen Patient T Cells and Hematopoietic Stem Cells von Bzhilyanskaya, Vera, Brault, Julie, Liu, Siyuan, Zea Vera, Andres F., Kozhushko, Nikita, Lawson, Amanda, Choi, Uimook, Clark, Aaron B., Meis, Ronald J., Ma, Michelle, Lazzarotto, Cicera, Tsai, Shengdar, Wu, Xiaolin, Dahl, Gary A., Bergerson, Jenna, Freeman, Alexandra F., Kleinstiver, Benjamin, Malech, Harry L, De Ravin, Suk See

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unified genetic, computational and experimental framework identifies functionally relevant residues of the homing endonuclease I-BmoI von Kleinstiver, Benjamin P, Fernandes, Andrew D, Gloor, Gregory B, Edgell, David R

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