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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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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Limitations of mouse models for sickle cell disease conferred by their human globin transgene configurations von Woodard, Kaitly J., Doerfler, Phillip A., Mayberry, Kalin D., Sharma, Akshay, Levine, Rachel, Yen, Jonathan, Valentine, Virginia, Palmer, Lance E., Valentine, Marc, Weiss, Mitchell J.

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A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition von Traxler, Elizabeth A, Yao, Yu, Wang, Yong-Dong, Woodard, Kaitly J, Kurita, Ryo, Nakamura, Yukio, Hughes, Jim R, Hardison, Ross C, Blobel, Gerd A, Li, Chunliang, Weiss, Mitchell J

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Adenosine Base Editing of γ-Globin Promoters Induces Fetal Hemoglobin and Inhibit Erythroid Sickling von Mayuranathan, Thiyagaraj, Yen, Jonathan S., Newby, Gregory A., Yao, Yu, Porter, Shaina N., Woodard, Kaitly J., Zhang, Jingjing, Mayberry, Kalin, Sharma, Akshay, preutt-Miller, Shondra M., Tisdale, John F., Tsai, Shengdar Q., Liu, David R., Weiss, Mitchell J.

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Base Editing Eliminates the Sickle Cell Mutation and Pathology in Hematopoietic Stem Cells Derived Erythroid Cells von Yen, Jonathan S, Newby, Gregory A., Mayuranathan, Thiyagaraj, Porter, Shaina N., Yao, Yu, Woodard, Kaitly J., Mayberry, Kalin, Everette, Kelcee, Zhang, Jingjing, Henderson, Jordana M, Mccaffrey, Anton P, Yen, Jonathan, Tisdale, John F., Tsai, Shengdar Q., Liu, David R., Weiss, Mitchell J.

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Base editing of hematopoietic 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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Base editing of haematopoietic stem cells rescues sickle cell disease in mice von Newby, Gregory, 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, Zhao, Kevin, Miller, Shannon, Wang, Tina, Koblan, Luke, McCaffrey, Anton P, Tisdale, John F, Kalfa, Theodosia, Pruett-Miller, Shondra M, Tsai, Shengdar, Weiss, Mitchell J, Liu, David

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A genome-editing strategy to treat [beta]-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition von Traxler, Elizabeth A, Yao, Yu, Wang, Yong-Dong, Woodard, Kaitly J, Kurita, Ryo, Nakamura, Yukio, Hughes, Jim R, Hardison, Ross C, Blobel, Gerd A, Li, Chunliang, Weiss, Mitchell J

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CRISPR-Cas9 Genome Editing of γ-Globin Promoters in Human Hematopoietic Stem Cells to Induce Erythrocyte Fetal Hemoglobin for Treatment of β-Hemoglobinopathies von Metais, Jean-Yves, Doerfler, Phillip A, Mayuranathan, Thiyagaraj, Bauer, Daniel E., Fowler, Stephanie, Hsieh, Matthew, Katta, Varun, Keriwala, Sagar, Lazzarotto, Cicera, Luk, Kevin, Neel, Michael, Perry, Scott, Porter, Shaina, Ryu, Byoung Y., Sharma, Akshay, Shea, Devlin, Peters, Samuel, Tisdale, John F., Wolfe, Scot A., Woodard, Kaitly Jensen, Uchida, Naoya, Wu, Yuxuan, Yao, Yu, Zeng, Jing, Pruett-Miller, Shondra M., Tsai, Shengdar Q., Weiss, Mitchell J.

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