Towards a new era in medicine: therapeutic genome editing

by Porteus, Matthew H.
Published 2015

A Look to Future Directions in Gene Therapy Research for Monogenic Diseases

by Porteus, Matthew H, Connelly, Jon P, Pruett, Shondra M
Published 2006

Design and Development of Artificial Zinc Finger Transcription Factors and Zinc Finger Nucleases to the hTERT Locus

by Wilson, Kimberly A, Chateau, Morgan L, Porteus, Matthew H
Published 2013

Zinc finger nucleases: custom-designed molecular scissors for genome engineering of plant and mammalian cells

by Durai, Sundar, Mani, Mala, Kandavelou, Karthikeyan, Wu, Joy, Porteus, Matthew H., Chandrasegaran, Srinivasan
Published 2005

Lentiviral and targeted cellular barcoding reveals ongoing clonal dynamics of cell lines in vitro and in vivo

by Porter, Shaina N, Baker, Lee C, Mittelman, David, Porteus, Matthew H
Published 2014

Nuclease-mediated gene editing by homologous recombination of the human globin locus

by Voit, Richard A., Hendel, Ayal, Pruett-Miller, Shondra M., Porteus, Matthew H.
Published 2014

Use of Genome Engineering to Create Patient Specific MLL Translocations in Primary Human Hematopoietic Stem and Progenitor Cells

by Breese, Erin H., Buechele, Corina, Dawson, Catherine, Cleary, Michael L., Porteus, Matthew H.
Published 2015

Gene/Cell Therapy Approaches for Immune Dysregulation Polyendocrinopathy Enteropathy X-Linked Syndrome

by Passerini, Laura, Santoni de Sio, Francesca R., Porteus, Matthew H., Bacchetta, Rosa
Published 2014

Expanding the Repertoire of Target Sites for Zinc Finger Nuclease-mediated Genome Modification

by Wilson, Kimberly A, McEwen, Abbye E, Pruett-Miller, Shondra M, Zhang, Jiuli, Kildebeck, Eric J, Porteus, Matthew H
Published 2013

A survey of ex vivo/in vitro transduction efficiency of mammalian primary cells and cell lines with Nine natural adeno-associated virus (AAV1-9) and one engineered adeno-associated...

by Ellis, Brian L, Hirsch, Matthew L, Barker, Jenny C, Connelly, Jon P, Steininger, Robert J, Porteus, Matthew H
Published 2013

Genome Editing in Mouse Spermatogonial Stem/Progenitor Cells Using Engineered Nucleases

by Fanslow, Danielle A., Wirt, Stacey E., Barker, Jenny C., Connelly, Jon P., Porteus, Matthew H., Dann, Christina Tenenhaus
Published 2014

Viral Single-Strand DNA Induces p53-Dependent Apoptosis in Human Embryonic Stem Cells

by Hirsch, Matthew L., Fagan, B. Matthew, Dumitru, Raluca, Bower, Jacquelyn J., Yadav, Swati, Porteus, Matthew H., Pevny, Larysa H., Samulski, R. Jude
Published 2011

Receptor-mediated delivery of engineered nucleases for genome modification

by Chen, Zhong, Jaafar, Lahcen, Agyekum, Davies G., Xiao, Haiyan, Wade, Marlene F., Kumaran, R. Ileng, Spector, David L., Bao, Gang, Porteus, Matthew H., Dynan, William S., Meiler, Steffen E.
Published 2013

SAPTA: a new design tool for improving TALE nuclease activity

by Lin, Yanni, Fine, Eli J., Zheng, Zhilan, Antico, Christopher J., Voit, Richard A., Porteus, Matthew H., Cradick, Thomas J., Bao, Gang
Published 2014

TALENs Facilitate Single-step Seamless SDF Correction of F508del CFTR in Airway Epithelial Submucosal Gland Cell-derived CF-iPSCs

by Suzuki, Shingo, Sargent, R Geoffrey, Illek, Beate, Fischer, Horst, Esmaeili-Shandiz, Alaleh, Yezzi, Michael J, Lee, Albert, Yang, Yanu, Kim, Soya, Renz, Peter, Qi, Zhongxia, Yu, Jingwei, Muench, Marcus O, Beyer, Ashley I, Guimarães, Alessander O, Ye, Lin, Chang, Judy, Fine, Eli J, Cradick, Thomas J, Bao, Gang, Rahdar, Meghdad, Porteus, Matthew H, Shuto, Tsuyoshi, Kai, Hirofumi, Kan, Yuet W, Gruenert, Dieter C
Published 2016

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

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