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Published in Science (American Association for the Advancement of Science) (25.03.2016)
Published in Science (American Association for the Advancement of Science) (25.03.2016)
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Gene Editing on Center Stage
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Published in Trends in genetics (01.08.2018)
Published in Trends in genetics (01.08.2018)
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Generation of human striatal organoids and cortico-striatal assembloids from human pluripotent stem cells
Miura, Yuki, Li, Min-Yin, Birey, Fikri, Ikeda, Kazuya, Revah, Omer, Thete, Mayuri Vijay, Park, Jin-Young, Puno, Alyssa, Lee, Samuel H, Porteus, Matthew H, Pașca, Sergiu P
Published in Nature biotechnology (01.12.2020)
Published in Nature biotechnology (01.12.2020)
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Identification of preexisting adaptive immunity to Cas9 proteins in humans
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
Published in Nature medicine (01.02.2019)
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A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells
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
Published in Nature medicine (01.08.2018)
Published in Nature medicine (01.08.2018)
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CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells
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
Published in Nature (London) (17.11.2016)
Published in Nature (London) (17.11.2016)
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Cell and gene therapy accessibility
Rouce, Rayne H., Porteus, Matthew H.
Published in Science (American Association for the Advancement of Science) (02.08.2024)
Published in Science (American Association for the Advancement of Science) (02.08.2024)
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Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice
Wilkinson, Adam C, Dever, Daniel P, Baik, Ron, Camarena, Joab, Hsu, Ian, Charlesworth, Carsten T, Morita, Chika, Nakauchi, Hiromitsu, Porteus, Matthew H
Published in Nature communications (29.01.2021)
Published in Nature communications (29.01.2021)
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Chimeric Nucleases Stimulate Gene Targeting in Human Cells
Porteus, Matthew H., Baltimore, David
Published in Science (American Association for the Advancement of Science) (02.05.2003)
Published in Science (American Association for the Advancement of Science) (02.05.2003)
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Gene correction for SCID-X1 in long-term hematopoietic stem cells
Pavel-Dinu, Mara, Wiebking, Volker, Dejene, Beruh T, Srifa, Waracharee, Mantri, Sruthi, Nicolas, Carmencita E, Lee, Ciaran, Bao, Gang, Kildebeck, Eric J, Punjya, Niraj, Sindhu, Camille, Inlay, Matthew A, Saxena, Nivedita, DeRavin, Suk See, Malech, Harry, Roncarolo, Maria Grazia, Weinberg, Kenneth I, Porteus, Matthew H
Published in Nature communications (09.04.2019)
Published in Nature communications (09.04.2019)
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Improving the safety of human pluripotent stem cell therapies using genome-edited orthogonal safeguards
Martin, Renata M, Fowler, Jonas L, Cromer, M Kyle, Lesch, Benjamin J, Ponce, Ezequiel, Uchida, Nobuko, Nishimura, Toshinobu, Porteus, Matthew H, Loh, Kyle M
Published in Nature communications (01.06.2020)
Published in Nature communications (01.06.2020)
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Highly Efficient and Marker-free Genome Editing of Human Pluripotent Stem Cells by CRISPR-Cas9 RNP and AAV6 Donor-Mediated Homologous Recombination
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.
Published in Cell stem cell (02.05.2019)
Published in Cell stem cell (02.05.2019)
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Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I
Gomez-Ospina, Natalia, Scharenberg, Samantha G, Mostrel, Nathalie, Bak, Rasmus O, Mantri, Sruthi, Quadros, Rolen M, Gurumurthy, Channabasavaiah B, Lee, Ciaran, Bao, Gang, Suarez, Carlos J, Khan, Shaukat, Sawamoto, Kazuki, Tomatsu, Shunji, Raj, Nitin, Attardi, Laura D, Aurelian, Laure, Porteus, Matthew H
Published in Nature communications (06.09.2019)
Published in Nature communications (06.09.2019)
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