Engineering monocyte/macrophage−specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing

por Samantha G. Scharenberg, Edina Poletto, Katherine L. Lucot, Pasqualina Colella, Adam Sheikali, Thomas J. Montine, Matthew H. Porteus, Natalia Gomez-Ospina
Publicado 2020

Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice

por Adam C. Wilkinson, Daniel P. Dever, Ron Baik, Joab Camarena, Ian Hsu, Carsten T. Charlesworth, Chika Morita, Hiromitsu Nakauchi, Matthew H. Porteus
Publicado 2021

Author Correction: Engineering monocyte/macrophage−specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing

por Samantha G. Scharenberg, Edina Poletto, Katherine L. Lucot, Pasqualina Colella, Adam Sheikali, Thomas J. Montine, Matthew H. Porteus, Natalia Gomez-Ospina
Publicado 2020

The TRACE-Seq method tracks recombination alleles and identifies clonal reconstitution dynamics of gene targeted human hematopoietic stem cells

por Rajiv Sharma, Daniel P. Dever, Ciaran M. Lee, Armon Azizi, Yidan Pan, Joab Camarena, Thomas Köhnke, Gang Bao, Matthew H. Porteus, Ravindra Majeti
Publicado 2021

Improving the safety of human pluripotent stem cell therapies using genome-edited orthogonal safeguards

por Renata M. Martin, Jonas L. Fowler, M. Kyle Cromer, Benjamin J. Lesch, Ezequiel Ponce, Nobuko Uchida, Toshinobu Nishimura, Matthew H. Porteus, Kyle M. Loh
Publicado 2020

Functional significance of U2AF1 S34F mutations in lung adenocarcinomas

por Mohammad S. Esfahani, Luke J. Lee, Young-Jun Jeon, Ryan A. Flynn, Henning Stehr, Angela B. Hui, Noriko Ishisoko, Eric Kildebeck, Aaron M. Newman, Scott V. Bratman, Matthew H. Porteus, Howard Y. Chang, Ash A. Alizadeh, Maximilian Diehn
Publicado 2019

Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I

por Natalia Gomez-Ospina, Samantha G. Scharenberg, Nathalie Mostrel, Rasmus O. Bak, Sruthi Mantri, Rolen M. Quadros, Channabasavaiah B. Gurumurthy, Ciaran Lee, Gang Bao, Carlos J. Suarez, Shaukat Khan, Kazuki Sawamoto, Shunji Tomatsu, Nitin Raj, Laura D. Attardi, Laure Aurelian, Matthew H. Porteus
Publicado 2019

Author Correction: Gene correction for SCID-X1 in long-term hematopoietic stem cells

por Mara Pavel-Dinu, Volker Wiebking, Beruh T. Dejene, Waracharee Srifa, Sruthi Mantri, Carmencita E. Nicolas, Ciaran Lee, Gang Bao, Eric J. Kildebeck, Niraj Punjya, Camille Sindhu, Matthew A. Inlay, Nivedita Saxena, Suk See DeRavin, Harry Malech, Maria Grazia Roncarolo, Kenneth I. Weinberg, Matthew H. Porteus
Publicado 2019

Gene correction for SCID-X1 in long-term hematopoietic stem cells

por Mara Pavel-Dinu, Volker Wiebking, Beruh T. Dejene, Waracharee Srifa, Sruthi Mantri, Carmencita E. Nicolas, Ciaran Lee, Gang Bao, Eric J. Kildebeck, Niraj Punjya, Camille Sindhu, Matthew A. Inlay, Nivedita Saxena, Suk See DeRavin, Harry Malech, Maria Grazia Roncarolo, Kenneth I. Weinberg, Matthew H. Porteus
Publicado 2019

Author Correction: Gene correction for SCID-X1 in long-term hematopoietic stem cells

por Mara Pavel-Dinu, Volker Wiebking, Beruh T. Dejene, Waracharee Srifa, Sruthi Mantri, Carmencita E. Nicolas, Ciaran Lee, Gang Bao, Eric J. Kildebeck, Niraj Punjya, Camille Sindhu, Matthew A. Inlay, Nivedita Saxena, Suk See DeRavin, Harry Malech, Maria Grazia Roncarolo, Kenneth I. Weinberg, Matthew H. Porteus
Publicado 2019

Mutations in the nuclear bile acid receptor FXR cause progressive familial intrahepatic cholestasis

por Natalia Gomez-Ospina, Carol J. Potter, Rui Xiao, Kandamurugu Manickam, Mi-Sun Kim, Kang Ho Kim, Benjamin L. Shneider, Jennifer L. Picarsic, Theodora A. Jacobson, Jing Zhang, Weimin He, Pengfei Liu, A. S. Knisely, Milton J. Finegold, Donna M. Muzny, Eric Boerwinkle, James R. Lupski, Sharon E. Plon, Richard A. Gibbs, Christine M. Eng, Yaping Yang, Gabriel C. Washington, Matthew H. Porteus, William E. Berquist, Neeraja Kambham, Ravinder J. Singh, Fan Xia, Gregory M. Enns, David D. Moore
Publicado 2016