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Yanfeng Li, Chandan Guha, Patrik Asp, Xia Wang, Tatyana L. Tchaikovskya, Kenneth Kim, Matthew Mendel, Gregory J. Cost, David H. Perlmutter, Namita Roy-Chowdhury, Ira J. Fox, Anthony Conway, Jayanta Roy-Chowdhury
Li, Y., Guha, C., Asp, P., et al. Resolution of hepatic fibrosis after zinc-finger nuclease-mediated gene editing in the PiZ mouse model of human alpha-1-antitrypsin deficiency liver disease (2023). https://journals.lww.com/hepcomm/Fulltext/2023/03010/ Resolution_of_hepatic_fibrosis_after_ZFN_mediated.23.aspx
Categories:
AAV, Genome Editing, Hepatic Fibrosis, Liver Disease, Publications, Zinc Finger Nuclease
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Paul Harmatz, Carlos E. Prada, Barbara K. Burton, Heather Lau, Craig M. Kessler, Liching Cao, Marina Falaleeva, Andres G. Villegas, Jennifer Zeitler, Kathleen Meyer, Weston Miller, Cheryl Wong Po Foo, Sagar Vaidya, Wendy Swenson, Lisa H. Shiue, Didier Rouy, Joseph Muenzer
Harmatz, P., Prada, C., Burton, B., et al. First-in-human in vivo genome editing via AAV-zinc finger nucleases for mucopolysaccharidosis I/II and hemophilia B, Mol Ther (2022). https://doi.org/10.1016/j.ymthe.2022.10.010
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AAV, Hemophilia B, In Vivo, MPS I, MPS II, Publications, Zinc Finger Nuclease
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Dr. Jeffrey S Moffit, Dr. Diann L Blanset, Dr. Jessica L Lynch, Dr. Timothy K MacLachlan, Dr. Kathleen E Meyer, Dr. Rafael Ponce, and Dr. Laurence Whiteley
Citation: Moffit, J., Blanset, D., Lynch, J., et al. Regulatory Consideration for the Nonclinical Safety Assessment of Gene Therapies. Mary Ann Liebert, Inc (2022). https://doi.org/10.1089/hum.2022.090
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Gene Therapy, Pediatric Safety, Publications, Regulatory, Reproduction
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Emma Proics, Marion David, Majid Mojibian, Madeline Speck, Nadia Lounnas-Mourey, Adeline Govehovitch, Wissam Baghdadi, Justine Desnouveaux, Hervé Bastian, Laura Freschi, Geoffrey Privat, Cédric Pouzet, Mauro Grossi, Pierre Heimendinger, Tobias Abel, David Fenard, Megan K Levings, François Meyer and Céline Dumont
Proics, E., David, M., Mojibian, M. et al. Preclinical assessment of antigen-specific chimeric antigen receptor regulatory T cells for use in solid organ transplantation. Gene Ther (2022). https://doi.org/10.1038/s41434-022-00358-x
Categories:
CAR-T, Organ Transplantation, Publications, Regulatory T Cells
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Liching Cao, Annemarie Ledeboer, Yonghua Pan, Yanmei Lu & Kathleen Meyer
Cao, L., Ledeboer, A., Pan, Y. et al. Clinical enrollment assay to detect preexisting neutralizing antibodies to AAV6 with demonstrated transgene expression in gene therapy trials. Gene Ther (2022). https://doi.org/10.1038/s41434-022-00353-2
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AAV6, Assay, Gene Therapy, Neutralizing Antibodies, Publications, Study Design
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Katharina Schreeb, Emily Culme-Seymour, Essra Ridha, Céline Dumont, Gillian Atkinson, Ben Hsu and Petra Reinke
Schreeb, K., Culme-Seymour, E., Ridha, E. et al., Study Design: Human Leukocyte Antigen Class I Molecule A* 02-Chimeric Antigen Receptor Regulatory T Cells in Renal Transplantation. Kidney Int Rep (2022) doi.org/10.1016/j.ekir.2022.03.030
Categories:
CAR-T, Cell Therapy, Clinical Trial, Publications, Renal Transplantation, Study Design
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Guangwen Wang, Pierre Heimendinger, Andrew Ramelmeier, Wenshi Wang
Wang, G., Heimendinger, P., Ramelmeier, A., et al. Pluripotent stem cell-based cell therapies: Current applications and future prospects. Curr Opin Biomed Eng (2022) doi.org/10.1016/j.cobme.2022.100390
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Allogeneic Cell Therapy, Hypoimmunogenic, Pluripotent Stem Cell, Publications
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J. Phillip Ramsey, Santoshkumar L. Khatwani, Min Lin, Rajeev Boregowda, Richard Surosky, R. Andrew Ramelmeier
Ramsey, J.P., Khatwani, S.L., Lin, M. et al. Overview of Analytics Needed to Support a Robust Gene Therapy Manufacturing Process. Curr Opin Biomed Eng (2021). doi: https://doi.org/10.1016/j.cobme.2021.100339
Categories:
AAV, Analytics, Gene Therapy, Manufacturing, Publications
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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
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AAV, Biochemistry, Genetic Engineering, Genome Editing, Publications, Technology
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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.
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Fabry Disease, Genome Editing, Liver, Lysosomal Storage Disorders, Preclinical Studies, Publications
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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
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Central Nervous System, Genome Regulation, Preclinical Studies, Publications
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Bart Corsaro, Tong-yuan Yang, Rocio Murphy, Ivo Sonderegger, Andrew Exley, Sylvie Bertholet, Naveen Dakappagari, Francis Dessy, Fabio Garofolo, Lisa Kierstead, Holger Koch, Ghanashyam Sarikonda, Natasha Savoie, Richard Siggers, Therese Solstad, Yanmei Lu, Mark Milton, Jean-Claude Marshall, Jason DelCarpini, Boris Gorovits, Swati Gupta, Lynne Jesaitis, John Kamerud, Arno Kromminga, Anna Ma, Jim McNally, Haoheng Yan, Bonnie Wu, Daniela Verthelyi, Susan Kirshner, Joao Pedras-Vasconcelos, Manoj Rajadhyaksha, Roland F. Staack, Elana Cherry, Isabelle Cludts, Madeleine Dahlbäck, George R. Gunn, Akiko Ishii-Watabe, Vibha Jawa, Robert Kubiak, Michael Partridge, Marco Petrillo, Samuel O. Pine, Johann Poetzl, Sam Song, Chris Stebbins, Yuling Wu, Lucia Zhang, Sumit Kar, Meina Liang, Mohsen Rajabi Abhari, Becky Schweighardt, Kay Stubenrauch & Yuanxin Xu
Corsaro, B., Yang, T., Murphy, R., et al. 2020 White Paper on Recent Issues in Bioanalysis: Vaccine Assay Validation, qPCR Assay Validation, QC for CAR-T Flow Cytometry, NAb Assay Harmonization and ELISpot Validation (Part 3 – Recommendations on Immunogenicity Assay Strategies, NAb Assays, Biosimilars and FDA/EMA Immunogenicity Guidance/Guideline, Gene & Cell Therapy and Vaccine Assays). Bioanalysis (2021) doi: https://doi.org/10.4155/bio-2021-0007
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Bioanalysis, Biomarkers, CAR-T, Cell Therapy, Gene Therapy, Immunogenicity, Publications, Vaccine
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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
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AAV, Fabry Disease, Gene Therapy, Liver, Lysosomal Storage Disorders, Preclinical Studies, Publications
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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
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Cell Therapy, Gene Targeting, Genome Editing, Publications, Zinc Finger Nuclease
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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
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Gene Targeting, Genome Editing, Publications, Zinc Finger Nuclease
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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
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Cell Therapy, Gene Targeting, Genome Editing, Publications, Zinc Finger Nuclease
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Jeffrey C. Miller, Deepak P. Patil, Danny F. Xia, Charles B. Paine, Friedrich Fauser, Hunter W. Richards, David A. Shivak, Yuri R. Bendaña, Sarah J. Hinkley, Nicholas A. Scarlott, Stephen C. Lam, Andreas Reik, Yuanyue Zhou, David E. Paschon, Patrick Li, Tenzin Wangzor, Gary Lee, Lei Zhang & Edward J. Rebar
Miller, J.C., Patil, D.P., Xia, D.F. et al. Enhancing gene editing specificity by attenuating DNA cleavage kinetics. Nat Biotechnol 37, 945–952 (2019) doi:10.1038/s41587-019-0186-z
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Biochemistry, Biotechnology, Enzyme Mechanisms, Gene Targeting, Publications, Zinc Finger Nuclease
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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
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Gene Silencing, Gene Therapy, Huntington's Disease, Publications
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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
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Biotechnology, Genetics, Protein Design, Publications, Zinc Finger Nuclease
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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
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Gene Therapy, Genetic Engineering, Publications
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Green F, Samaranch L, Zhang HS, Manning-Bog A, Meyer K, Forsayeth J, Bankiewicz KS.
Gene Ther. 2016 Jun;23(6):520-6. doi: 10.1038/gt.2016.24. Epub 2016 Mar 8.
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Gene Therapy, Publications, Technology
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Li H, Haurigot V, Doyon Y, Li T, Wong SY, Bhagwat AS, Malani N, Anguela XM, Sharma R, Ivanciu L, Murphy SL, Finn JD, Khazi FR, Zhou S, Paschon DE, Rebar EJ, Bushman FD, Gregory PD, Holmes MC, High KA.
Nature. 2011 Jun 26;475(7355):217-21. doi: 10.1038/nature10177.
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Genome Editing, Hematology, Publications
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Haemophilia. 2016 Jul;22 Suppl 5:66-71. doi: 10.1111/hae.13011. Review.
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Gene Therapy, Hematology, Publications
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Holt N, Wang J, Kim K, Friedman G, Wang X, Taupin V, Crooks GM, Kohn DB, Gregory PD, Holmes MC, Cannon PM.
Nat Biotechnol. 2010 Aug;28(8):839-47. doi: 10.1038/nbt.1663. Epub 2010 Jul 2.
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Cell Therapy, Infectious Disease (HIV/AIDS), Publications
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Nat Methods. 2015 Oct;12(10):927-30. doi: 10.1038/nmeth.3554. Epub 2015 Aug 31.
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Cell Therapy, Hematology, Publications, Technology
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Wang J, DeClercq JJ, Hayward SB, Li PW, Shivak DA, Gregory PD, Lee G, Holmes MC.
Nucleic Acids Res. 2016 Feb 18;44(3):e30. doi: 10.1093/nar/gkv1121. Epub 2015 Nov 2.
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Cell Therapy, Immunology, Oncology, Publications, Technology
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Wang J, Exline CM, DeClercq JJ, Llewellyn GN, Hayward SB, Li PW, Shivak DA, Surosky RT, Gregory PD, Holmes MC, Cannon PM.
Nat Biotechnol. 2015 Dec;33(12):1256-1263. doi: 10.1038/nbt.3408. Epub 2015 Nov 9.
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Cell Therapy, Publications, Technology
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Christine E Brown, Analiz Rodriguez, Joycelynne Palmer, Julie R Ostberg, Araceli Naranjo, Jamie R Wagner, Brenda Aguilar, Renate Starr, Lihong Weng, Timothy W Synold, Vivi Tran, Shelley Wang, Andreas Reik, Massimo D’Apuzzo, Julie A Ressler, Yuanyue Zhou, Matthew Mendel, Philip D Gregory, Michael C Holmes, Winson W Tang, Stephen J Forman, Michael C Jensen, Behnam Badie
Brown, C.E, Rodriguez, A., Palmer, J., Ostberg, J.R. et al., Off-the-shelf, steroid-resistant, IL13Rα2-specific CAR T cells for treatment of glioblastoma, Neuro-Oncology, 2022; noac024, https://doi.org/10.1093/neuonc/noac024
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CAR-T, Cell Therapy, Clinical Trial, Glioblastoma, Oncology, Publications, Zinc Finger Nuclease
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Cytotherapy. 2016 Nov;18(11):1370-1381. doi: 10.1016/j.jcyt.2016.07.007. Review.
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Cell Therapy, Infectious Disease (HIV/AIDS), Publications
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Peterson CW, Wang J, Norman KK, Norgaard ZK, Humbert O, Tse CK, Yan JJ, Trimble RG, Shivak DA, Rebar EJ, Gregory PD, Holmes MC, Kiem HP.
Blood. 2016 May 19;127(20):2416-26. doi: 10.1182/blood-2015-09-672337. Epub 2016 Mar 15.
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Cell Therapy, Infectious Disease (HIV/AIDS), Publications
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Miller JC, Holmes MC, Wang J, Guschin DY, Lee YL, Rupniewski I, Beausejour CM, Waite AJ, Wang NS, Kim KA, Gregory PD, Pabo CO, Rebar EJ.
Nat Biotechnol. 2007 Jul;25(7):778-85. Epub 2007 Jul 1.
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Genome Editing, Publications, Technology
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Urnov FD, Miller JC, Lee YL, Beausejour CM, Rock JM, Augustus S, Jamieson AC, Porteus MH, Gregory PD, Holmes MC.
Nature. 2005 Jun 2;435(7042):646-51. Epub 2005 Apr 3.
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Genome Editing, Publications, Technology
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Tan S, Guschin D, Davalos A, Lee YL, Snowden AW, Jouvenot Y, Zhang HS, Howes K, McNamara AR, Lai A, Ullman C, Reynolds L, Moore M, Isalan M, Berg LP, Campos B, Qi H, Spratt SK, Case CC, Pabo CO, Campisi J, Gregory PD.
Proc Natl Acad Sci U S A. 2003 Oct 14;100(21):11997-2002. Epub 2003 Sep 26.
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Genome Regulation, Publications, Technology
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Chang KH, Smith SE, Sullivan T, Chen K, Zhou Q, West JA, Liu M, Liu Y, Vieira BF, Sun C, Hong VP, Zhang M, Yang X, Reik A, Urnov FD, Rebar EJ, Holmes MC, Danos O, Jiang H, Tan S.
Mol Ther Methods Clin Dev. 2017 Jan 11;4:137-148. doi: 10.1016/j.omtm.2016.12.009. eCollection 2017 Mar 17.
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Cell Therapy, Hematology, Publications
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Laoharawee K, DeKelver RC, Podetz-Pedersen KM, Rohde M, Sproul S, Nguyen HO, Nguyen T, St Martin SJ, Ou L, Tom S, Radeke R, Meyer KE, Holmes MC, Whitley CB, Wechsler T, McIvor RS.
Mol Ther. 2018 Apr 4;26(4):1127-1136. doi: 10.1016/j.ymthe.2018.03.002. Epub 2018 Mar 10.
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Genome Editing, Inherited Metabolic Diseases, Publications
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Tebas P, Stein D, Tang WW, Frank I, Wang SQ, Lee G, Spratt SK, Surosky RT, Giedlin MA, Nichol G, Holmes MC, Gregory PD, Ando DG, Kalos M, Collman RG, Binder-Scholl G, Plesa G, Hwang WT, Levine BL, June CH.
N Engl J Med. 2014 Mar 6;370(10):901-10. doi: 10.1056/NEJMoa1300662.
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Cell Therapy, Infectious Disease (HIV/AIDS), Publications
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Davidoff AM, Nathwani AC.
N Engl J Med. 2016 Mar 31;374(13):1288-90. doi: 10.1056/NEJMcibr1600347.
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Genome Editing, Hematology, Publications
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Urnov FD, Rebar EJ, Holmes MC, Zhang HS, Gregory PD.
Nat Rev Genet. 2010 Sep;11(9):636-46. doi: 10.1038/nrg2842. Review.
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Genome Editing, Publications, Technology
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Torikai H, Reik A, Soldner F, Warren EH, Yuen C, Zhou Y, Crossland DL, Huls H, Littman N, Zhang Z, Tykodi SS, Kebriaei P, Lee DA, Miller JC, Rebar EJ, Holmes MC, Jaenisch R, Champlin RE, Gregory PD, Cooper LJ.
Blood. 2013 Aug 22;122(8):1341-9. doi: 10.1182/blood-2013-03-478255. Epub 2013 Jun 5.
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Cell Therapy, Publications
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Torikai H, Reik A, Liu PQ, Zhou Y, Zhang L, Maiti S, Huls H, Miller JC, Kebriaei P, Rabinovich B, Lee DA, Champlin RE, Bonini C, Naldini L, Rebar EJ, Gregory PD, Holmes MC, Cooper LJ.
Blood. 2012 Jun 14;119(24):5697-705. doi: 10.1182/blood-2012-01-405365. Epub 2012 Apr 24.
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Cell Therapy, Oncology, Publications