sangamo publications

Our robust collection of publications, authored by our own scientists, collaborators, and others, contains original research articles and review articles that are relevant to our ZFP technology and its many applications.

Anion-exchange HPLC assay for Separation and Quantification of Empty and Full Capsids in Multiple Adeno-associated Virus Serotypes

Santoshkumar L. Khatwani, Anna Pavlova, Zhu Pirot

Khatwani, S.L., Pavlova, A., Pirot, Z. Anion-exchange HPLC assay for Separation and Quantification of Empty and Full Capsids in Multiple Adeno-associated Virus Serotypes. Molecular Therapy: Methods & Clinical Development (2021) doi: https://doi.org/10.1016/j.omtm.2021.04.003

Categories: Genome Editing, Gene Therapy, Technology, Genetic Engineering, Biotechnology, AAV

ZFN-Mediated In Vivo Gene Editing in Hepatocytes Leads to Supraphysiologic α-Gal A Activity and Effective Substrate Reduction in Fabry Mice

Silvere Pagant, Marshall W. Huston , Luciana Moreira, Lin Gan, Susan St. Martin, Scott Sproul, Michael C. Holmes, Kathleen Meyer, Thomas Wechsler, Robert J. Desnick, Makiko Yasuda

Pagant, S., Huston, M. W., Moreira, L. et al. ZFN-Mediated In Vivo Gene Editing in Hepatocytes Leads to Supraphysiologic α-Gal A Activity and Effective Substrate Reduction in Fabry Mice. Mol Ther (2021) doi: https://doi.org/10.1016/j.ymthe.2021.03.018.

Categories: Genome Editing, Lysosomal Storage Disorders, Fabry Disease, Preclinical Studies, Liver

Persistent repression of tau in the brain using engineered zinc finger protein transcription factors

Susanne Wegmann, Sarah L. DeVos, Bryan Zeitler, Kimberly Marlen, Rachel E. Bennett, Marta Perez-Rando, Danny MacKenzie, Qi Yu, Caitlin Commins, Riley N. Bannon, Bianca T. Corjuc, Alison Chase, Lisa Diez, Hoang-Oanh B. Nguyen, Sarah Hinkley, Lei Zhang, Alicia Goodwin, Annemarie Ledeboer, Stephen Lam, Irina Ankoudinova, Hung Tran, Nicholas Scarlott, Rainier Amora, Richard Surosky, Jeffrey C. Miller, Ashley B. Robbins, Edward J. Rebar, Fyodor D. Urnov, Michael C. Holmes, Amy M. Pooler, Brigit Riley, H. Steve Zhang, Bradley T. Hyman

Wegmann, S., DeVos, S. L., Zeitler, B. et al. Persistent repression of tau in the brain using engineered zinc finger protein transcription factors. Sci. Adv. 7, eabe1611 (2021) doi: 0.1126/sciadv.abe1611

Categories: Genome Regulation, Central Nervous System, Preclinical Studies

AAV2/6 Gene Therapy in a Murine Model of Fabry Disease Results in Supraphysiological Enzyme Activity and Effective Substrate Reduction

Makiko Yasuda, Marshall W. Huston, Silvere Pagant, Lin Gan, Susan St. Martin, Scott Sproul, Daniel Richards, Stephen Ballaron, Khaled Hettini, Annemarie Ledeboer, Lillian Falese, Liching Cao, Yanmei Lu, Michael C. Holmes, Kathleen Meyer, Robert J. Desnick, Thomas Wechsler

Yasuda, M., Huston, M.W., Pagant, S. et al. AAV2/6 gene therapy in a murine model of Fabry disease results in supraphysiological enzyme activity and effective substrate reduction. Molecular Therapy: Methods & Clinical Development, P607-619 (2020) doi:10.1016/j.omtm.2020.07.002

Categories: Gene Therapy, AAV, Lysosomal Storage Disorders, Fabry Disease, Preclinical Studies, Liver

Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1

Giulia Schiroli, Samuele Ferrari, Anthony Conway, Aurelien Jacob, Valentina Capo, Luisa Albano, Tiziana Plati, Maria C. Castiello, Francesca Sanvito, Andrew R. Gennery, Chiara Bovolenta, Rahul Palchaudhuri, David T. Scadden, Michael C. Holmes, Anna Villa, Giovanni Sitia, Angelo Lombardo, Pietro Genovese & Luigi Naldini

Schiroli, G., Ferrari, S., Conway, A. et al. Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1. Science Translational Medicine 9, eaan0820 (2017) doi: 10.1126/scitranslmed.aan0820

Categories: Genome Editing, Cell Therapy, Zinc Finger Nuclease, Gene Targeting

Non-viral Delivery of Zinc Finger Nuclease mRNA Enables Highly Efficient In Vivo Genome Editing of Multiple Therapeutic Gene Targets

Anthony Conway, Matthew Mendel, Kenneth Kim, Kyle McGovern, Alisa Boyko, Lei Zhang, Jeffrey C. Miller, Russell C. DeKelver, David E. Paschon, Barbara L. Mui, Paulo J.C. Lin, Ying K. Tam, Chris Barbosa, Tom Redelmeier, Michael C. Holmes & Gary Lee

Conway, A., Mendel, M., Kim, K. et al. Non-viral Delivery of Zinc Finger Nuclease mRNA Enables Highly Efficient In Vivo Genome Editing of Multiple Therapeutic Gene Targets. Mol Ther 27, P866-877 (2019) doi: 10.1016/j.ymthe.2019.03.003

Categories: Genome Editing, Zinc Finger Nuclease, Gene Targeting

Highly Efficient Gene Editing of Cystic Fibrosis Patient-derived Airway Basal Cells Results in Functional CFTR Correction

Shingo Suzuki, Ana M. Crane, Varada Anirudhan, Cristina Barillà, Nadine Matthias, Scott H. Randell, Andras Rab, Eric J. Sorscher, Jenny L. Kerschner, Shiyi Yin, Ann Harris, Matthew Mendel, Kenneth Kim, Lei Zhang, Anthony Conway & Brian R. Davis

Suzuki, S., Crane, A.M., Anirudhan, V. et al. Highly Efficient Gene Editing of Cystic Fibrosis Patient-derived Airway Basal Cells Results in Functional CFTR Correction. Mol Ther (2020) doi: 10.1016/j.ymthe.2020.04.021

Categories: Genome Editing, Cell Therapy, Zinc Finger Nuclease, Gene Targeting

Genome editing in mitochondria corrects a pathogenic mtDNA mutation in vivo

Payam A. Gammage, Carlo Viscomi, Marie-Lune Simard, Ana S. H. Costa, Edoardo Gaude, Christopher A. Powell, Lindsey Van Haute, Beverly J. McCann, Pedro Rebelo-Guiomar, Raffaele Cerutti, Lei Zhang, Edward J. Rebar, Massimo Zeviani, Christian Frezza, James B. Stewart & Michal Minczuk

Gammage, P.A., Viscomi, C., Simard, M. et al. Genome editing in mitochondria corrects a pathogenic mtDNA mutation in vivo. Nat Med 24, 1691–1695 (2018) doi:10.1038/s41591-018-0165-9

Categories: Gene Therapy, Genetic Engineering

Diversifying the structure of zinc finger nucleases for high-precision genome editing

David E. Paschon, Stephanie Lussier, Tenzin Wangzor, Danny F. Xia, Patrick W. Li, Sarah J. Hinkley, Nicholas A. Scarlott, Stephen C. Lam, Adam J. Waite, Lynn N. Truong, Nimisha Gandhi, Bhakti N. Kadam, Deepak P. Patil, David A. Shivak, Gary K. Lee, Michael C. Holmes, Lei Zhang, Jeffrey C. Miller & Edward J. Rebar

Paschon, D.E., Lussier, S., Wangzor, T. et al. Diversifying the structure of zinc finger nucleases for high-precision genome editing. Nat Commun 10, 1133 (2019) doi:10.1038/s41467-019-08867-x

Categories: Biotechnology, Genetics, Protein Design, Zinc Finger Nuclease

Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington’s disease

Bryan Zeitler, Steven Froelich, Kimberly Marlen, David A Shivak, Qi Yu, Davis Li, Jocelynn R Pearl, Jeffrey C Miller, Lei Zhang, David E Paschon, Sarah J Hinkley, Irina Ankoudinova, Stephen Lam, Dmitry Guschin, Lexi Kopan, Jennifer M Cherone, Hoang-Oanh B Nguyen, Guijuan Qiao, Yasaman Ataei, Matthew C Mendel, Rainier Amora, Richard Surosky, Josee Laganiere, B Joseph Vu, Anand Narayanan, Yalda Sedaghat, Karsten Tillack, Christina Thiede, Annette Gärtner, Seung Kwak, Jonathan Bard, Ladislav Mrzljak, Larry Park, Taneli Heikkinen, Kimmo K Lehtimäki, Marie M Svedberg, Jenny Häggkvist, Lenke Tari, Miklós Tóth, Andrea Varrone, Christer Halldin, Andrea E Kudwa, Sylvie Ramboz, Michelle Day, Jyothisri Kondapalli, D James Surmeier, Fyodor D Urnov, Philip D Gregory, Edward J Rebar, Ignacio Muñoz-Sanjuán & H Steve Zhang

Zeitler, B., Froelich, S., Marlen, K. et al. Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington’s disease. Nat Med 25, 1131–1142 (2019) doi:10.1038/s41591-019-0478-3

Categories: Gene Therapy, Gene Silencing, Huntington's disease