Base editing of haematopoietic stem cells rescues sickle cell disease in mice von Newby, Gregory A., Yen, Jonathan S., Woodard, Kaitly J., Mayuranathan, Thiyagaraj, Lazzarotto, Cicera R., Li, Yichao, Sheppard-Tillman, Heather, Porter, Shaina N., Yao, Yu, Mayberry, Kalin, Everette, Kelcee A., Jang, Yoonjeong, Podracky, Christopher J., Thaman, Elizabeth, Lechauve, Christophe, Sharma, Akshay, Henderson, Jordana M., Richter, Michelle F., Zhao, Kevin T., Miller, Shannon M., Wang, Tina, Koblan, Luke W., McCaffrey, Anton P., Tisdale, John F., Kalfa, Theodosia A., Pruett-Miller, Shondra M., Tsai, Shengdar Q., Weiss, Mitchell J., Liu, David R.

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Highly efficient therapeutic gene editing of human hematopoietic stem cells von Wu, Yuxuan, Zeng, Jing, Roscoe, Benjamin P., Liu, Pengpeng, Yao, Qiuming, Lazzarotto, Cicera R., Clement, Kendell, Cole, Mitchel A., Luk, Kevin, Baricordi, Cristina, Shen, Anne H., Ren, Chunyan, Esrick, Erica B., Manis, John P., Dorfman, David M., Williams, David A., Biffi, Alessandra, Brugnara, Carlo, Biasco, Luca, Brendel, Christian, Pinello, Luca, Tsai, Shengdar Q., Wolfe, Scot A., Bauer, Daniel E.

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Prime editing in mice reveals the essentiality of a single base in driving tissue-specific gene expression von Gao, Pan, Lyu, Qing, Ghanam, Amr R, Lazzarotto, Cicera R, Newby, Gregory A, Zhang, Wei, Choi, Mihyun, Slivano, Orazio J, Holden, Kevin, Walker, 2nd, John A, Kadina, Anastasia P, Munroe, Rob J, Abratte, Christian M, Schimenti, John C, Liu, David R, Tsai, Shengdar Q, Long, Xiaochun, Miano, Joseph M

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In vivo engineered B cells secrete high titers of broadly neutralizing anti-HIV antibodies in mice von Nahmad, Alessio D., Lazzarotto, Cicera R., Zelikson, Natalie, Kustin, Talia, Tenuta, Mary, Huang, Deli, Reuveni, Inbal, Nataf, Daniel, Raviv, Yuval, Horovitz-Fried, Miriam, Dotan, Iris, Carmi, Yaron, Rosin-Arbesfeld, Rina, Nemazee, David, Voss, James E., Stern, Adi, Tsai, Shengdar Q., Barzel, Adi

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Effects of imposed and self-selected exercise on perceptual and affective responses, muscle function, quality, and functionality of strength training in older women and men: a rand... von Garcia, E D S A, Ferreira, S S, Lazzarotto, R, Silva, J K F da, Bento, P C B

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The transgenic animal platform for biopharmaceutical production von Bertolini, L. R., Meade, H., Lazzarotto, C. R., Martins, L. T., Tavares, K. C., Bertolini, M., Murray, J. D.

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Genome editing of HBG1 and HBG2 to induce fetal hemoglobin von Métais, Jean-Yves, Doerfler, Phillip A., Mayuranathan, Thiyagaraj, Bauer, Daniel E., Fowler, Stephanie C., Hsieh, Matthew M., Katta, Varun, Keriwala, Sagar, Lazzarotto, Cicera R., Luk, Kevin, Neel, Michael D., Perry, S. Scott, Peters, Samuel T., Porter, Shaina N., Ryu, Byoung Y., Sharma, Akshay, Shea, Devlin, Tisdale, John F., Uchida, Naoya, Wolfe, Scot A., Woodard, Kaitly J., Wu, Yuxuan, Yao, Yu, Zeng, Jing, Pruett-Miller, Shondra, Tsai, Shengdar Q., Weiss, Mitchell J.

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CRISPR-Cas9-AAV versus lentivector transduction for genome modification of X-linked severe combined immunodeficiency hematopoietic stem cells von Brault, Julie, Liu, Taylor, Liu, Siyuan, Lawson, Amanda, Choi, Uimook, Kozhushko, Nikita, Bzhilyanskaya, Vera, Pavel-Dinu, Mara, Meis, Ronald J, Eckhaus, Michael A, Burkett, Sandra S, Bosticardo, Marita, Kleinstiver, Benjamin P, Notarangelo, Luigi D, Lazzarotto, Cicera R, Tsai, Shengdar Q, Wu, Xiaolin, Dahl, Gary A, Porteus, Matthew H, Malech, Harry L, De Ravin, Suk See

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Production and characterization of virus-free, CRISPR-CAR T cells capable of inducing solid tumor regression von Mueller, Katherine P, Piscopo, Nicole J, Forsberg, Matthew H, Saraspe, Louise A, Das, Amritava, Russell, Brittany, Smerchansky, Madeline, Cappabianca, Dan, Shi, Lei, Shankar, Keerthana, Sarko, Lauren, Khajanchi, Namita, La Vonne Denne, Nina, Ramamurthy, Apoorva, Ali, Adeela, Lazzarotto, Cicera R, Tsai, Shengdar Q, Capitini, Christian M, Saha, Krishanu

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CHANGE-seq reveals genetic and epigenetic effects on CRISPR–Cas9 genome-wide activity von Lazzarotto, Cicera R., Malinin, Nikolay L., Li, Yichao, Zhang, Ruochi, Yang, Yang, Lee, GaHyun, Cowley, Eleanor, He, Yanghua, Lan, Xin, Jividen, Kasey, Katta, Varun, Kolmakova, Natalia G., Petersen, Christopher T., Qi, Qian, Strelcov, Evgheni, Maragh, Samantha, Krenciute, Giedre, Ma, Jian, Cheng, Yong, Tsai, Shengdar Q.

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Genetic Inactivation of CD33 in Hematopoietic Stem Cells to Enable CAR T Cell Immunotherapy for Acute Myeloid Leukemia von Kim, Miriam Y., Yu, Kyung-Rok, Kenderian, Saad S., Ruella, Marco, Chen, Shirley, Shin, Tae-Hoon, Aljanahi, Aisha A., Schreeder, Daniel, Klichinsky, Michael, Shestova, Olga, Kozlowski, Miroslaw S., Cummins, Katherine D., Shan, Xinhe, Shestov, Maksim, Bagg, Adam, Morrissette, Jennifer J.D., Sekhri, Palak, Lazzarotto, Cicera R., Calvo, Katherine R., Kuhns, Douglas B., Donahue, Robert E., Behbehani, Gregory K., Tsai, Shengdar Q., Dunbar, Cynthia E., Gill, Saar

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Defining genome-wide CRISPR–Cas genome-editing nuclease activity with GUIDE-seq von Malinin, Nikolay L., Lee, GaHyun, Lazzarotto, Cicera R., Li, Yichao, Zheng, Zongli, Nguyen, Nhu T., Liebers, Matthew, Topkar, Ved V., Iafrate, A. John, Le, Long P., Aryee, Martin J., Joung, J. Keith, Tsai, Shengdar Q.

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Defining CRISPR–Cas9 genome-wide nuclease activities with CIRCLE-seq von Lazzarotto, Cicera R., Nguyen, Nhu T., Tang, Xing, Malagon-Lopez, Jose, Guo, Jimmy A., Aryee, Martin J., Joung, J. Keith, Tsai, Shengdar Q.

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Potent and uniform fetal hemoglobin induction via base editing von Mayuranathan, Thiyagaraj, Newby, Gregory A., Feng, Ruopeng, Yao, Yu, Mayberry, Kalin D., Lazzarotto, Cicera R., Li, Yichao, Levine, Rachel M., Nimmagadda, Nikitha, Dempsey, Erin, Kang, Guolian, Porter, Shaina N., Doerfler, Phillip A., Zhang, Jingjing, Jang, Yoonjeong, Chen, Jingjing, Bell, Henry W., Crossley, Merlin, Bhoopalan, Senthil Velan, Sharma, Akshay, Tisdale, John F., Pruett-Miller, Shondra M., Cheng, Yong, Tsai, Shengdar Q., Liu, David R., Weiss, Mitchell J., Yen, Jonathan S.

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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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BCL11A enhancer-edited hematopoietic stem cells persist in rhesus monkeys without toxicity von Demirci, Selami, Zeng, Jing, Wu, Yuxuan, Uchida, Naoya, Shen, Anne H, Pellin, Danilo, Gamer, Jackson, Yapundich, Morgan, Drysdale, Claire, Bonanno, Jasmine, Bonifacino, Aylin C, Krouse, Allen E, Linde, Nathaniel S, Engels, Theresa, Donahue, Robert E, Haro-Mora, Juan J, Leonard, Alexis, Nassehi, Tina, Luk, Kevin, Porter, Shaina N, Lazzarotto, Cicera R, Tsai, Shengdar Q, Weiss, Mitchell J, Pruett-Miller, Shondra M, Wolfe, Scot A, Bauer, Daniel E, Tisdale, John F

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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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Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease von Katta, Varun, O’Keefe, Kiera, Li, Yichao, Mayuranathan, Thiyagaraj, Lazzarotto, Cicera R., Wood, Rachael K., Levine, Rachel M., Powers, Alicia, Mayberry, Kalin, Manquen, Garret, Yao, Yu, Zhang, Jingjing, Jang, Yoonjeong, Nimmagadda, Nikitha, Dempsey, Erin A., Lee, GaHyun, Uchida, Naoya, Cheng, Yong, Fazio, Frank, Lockey, Tim, Meagher, Mike, Sharma, Akshay, Tisdale, John F., Zhou, Sheng, Yen, Jonathan S., Weiss, Mitchell J., Tsai, Shengdar Q.

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Prediction and validation of hematopoietic stem and progenitor cell off-target editing in transplanted rhesus macaques von AlJanahi, Aisha A., Lazzarotto, Cicera R., Chen, Shirley, Shin, Tae-Hoon, Cordes, Stefan, Fan, Xing, Jabara, Isabel, Zhou, Yifan, Young, David J., Lee, Byung-Chul, Yu, Kyung-Rok, Li, Yuesheng, Toms, Bradley, Tunc, Ilker, Hong, So Gun, Truitt, Lauren L., Klermund, Julia, Andrieux, Geoffroy, Kim, Miriam Y., Cathomen, Toni, Gill, Saar, Tsai, Shengdar Q., Dunbar, Cynthia E.

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BCL11A +58/+55 enhancer-editing facilitates HSPC engraftment and HbF induction in rhesus macaques conditioned with a CD45 antibody-drug conjugate von Demirci, Selami, Zeng, Jing, Palchaudhuri, Rahul, Wu, Chuanfeng, Abraham, Diana M, Hayal, Taha B, Essawi, Khaled, Nguyen, My Anh, Stasula, Ulana, Chu, Rebecca, Leonard, Alexis, Porter, Shaina N, Khan, Muhammad Behroz Naeem, Hinojosa, Gabriela, Uchida, Naoya, Hong, Sogun, Lazzarotto, Cicera R, Neri, Nola R, da Silva, Lucas Ferreira, Pellin, Danilo, Verma, Archana, Lanieri, Leanne, Bhat, Anjali, Hammond, Katelyn, Tate, Tiffany, Maitland, Stacy A, Sheikhsaran, Fatemeh, Bonifacino, Aylin C, Krouse, Allen E, Linde, Nathaniel S, Engels, Theresa, Golomb, Justin, Tsai, Shengdar Q, Pruett-Miller, Shondra M, Scadden, David T, Dunbar, Cynthia E, Wolfe, Scot A, Donahue, Robert E, Olson, Lisa M, Bauer, Daniel E, Tisdale, John F

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