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Editorial

Activating human genes with zinc finger proteins, transcription activator-like effectors and CRISPR/Cas9 for gene therapy and regenerative medicine

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Christian Huisman, Monique GP van der Wijst, Fahimeh Falahi, Juul Overkamp, Gellért Karsten, Martijn M Terpstra, Klaas Kok, Ate GJ van der Zee, Ed Schuuring, G Bea A Wisman & Marianne G Rots. (2015) Prolonged re-expression of the hypermethylated gene EPB41L3 using artificial transcription factors and epigenetic drugs. Epigenetics 10:5, pages 384-396.
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M Ian Phillips & Andrew B Burns. (2014) The emergence of gene therapy for rare diseases. Expert Opinion on Orphan Drugs 2:11, pages 1197-1209.
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Rachel Dennison, Esteban Usuga, Harriet Chen, Jacob Z. Paul, Christian A. Arbelaez & Yang D. Teng. (2023) Direct Cell Reprogramming and Phenotypic Conversion: An Analysis of Experimental Attempts to Transform Astrocytes into Neurons in Adult Animals. Cells 12:4, pages 618.
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Elvir Becirovic. (2022) Maybe you can turn me on: CRISPRa-based strategies for therapeutic applications. Cellular and Molecular Life Sciences 79:2.
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Niloofar FarhangBryton DavisJacob WestonMatthew Ginley-HidingerJason GertzRobby D. Bowles. (2020) Synergistic CRISPRa-Regulated Chondrogenic Extracellular Matrix Deposition Without Exogenous Growth Factors. Tissue Engineering Part A 26:21-22, pages 1169-1179.
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Jules J. Berman. 2020. Logic and Critical Thinking in the Biomedical Sciences. Logic and Critical Thinking in the Biomedical Sciences 187 208 .
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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. (2019) Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington’s disease. Nature Medicine 25:7, pages 1131-1142.
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Valentin Baumann, Maximilian Wiesbeck, Christopher T. Breunig, Julia M. Braun, Anna Köferle, Jovica Ninkovic, Magdalena Götz & Stefan H. Stricker. (2019) Targeted removal of epigenetic barriers during transcriptional reprogramming. Nature Communications 10:1.
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Jules J. Berman. 2019. Taxonomic Guide to Infectious Diseases. Taxonomic Guide to Infectious Diseases 19 37 .
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Ci-Di Chen, Ella Zeldich, Yuexuan Li, Andrea Yuste & Carmela R. Abraham. (2018) Activation of the Anti-Aging and Cognition-Enhancing Gene Klotho by CRISPR-dCas9 Transcriptional Effector Complex. Journal of Molecular Neuroscience 64:2, pages 175-184.
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Tobias Anton, Elisabeth Karg & Sebastian Bultmann. (2018) Applications of the CRISPR/Cas system beyond gene editing. Biology Methods and Protocols 3:1.
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Jules J. Berman. 2018. Precision Medicine and the Reinvention of Human Disease. Precision Medicine and the Reinvention of Human Disease 327 365 .
Anne-Sophie Gautron, Alexandre Juillerat, Valérie Guyot, Jean-Marie Filhol, Emilie Dessez, Aymeric Duclert, Philippe Duchateau & Laurent Poirot. (2017) Fine and Predictable Tuning of TALEN Gene Editing Targeting for Improved T Cell Adoptive Immunotherapy. Molecular Therapy - Nucleic Acids 9, pages 312-321.
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Nixon Niyonzima, Abigail R. Lambert, Rachel Werther, Harshana De Silva Feelixge, Pavitra Roychoudhury, Alexander L. Greninger, Daniel Stone, Barry L. Stoddard & Keith R. Jerome. (2017) Tuning DNA binding affinity and cleavage specificity of an engineered gene-targeting nuclease via surface display, flow cytometry and cellular analyses. Protein Engineering, Design and Selection 30:7, pages 503-522.
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Joshua B. Black, Pablo Perez-Pinera & Charles A. Gersbach. (2017) Mammalian Synthetic Biology: Engineering Biological Systems. Annual Review of Biomedical Engineering 19:1, pages 249-277.
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Alexander Brown, Wendy S. Woods & Pablo Perez-Pinera. 2017. Enhancer RNAs. Enhancer RNAs 235 250 .
Manjunath N. Swamy, Haoquan Wu & Premlata Shankar. (2016) Recent advances in RNAi-based strategies for therapy and prevention of HIV-1/AIDS. Advanced Drug Delivery Reviews 103, pages 174-186.
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Elena Matsa, John H. Ahrens & Joseph C. Wu. (2016) Human Induced Pluripotent Stem Cells as a Platform for Personalized and Precision Cardiovascular Medicine. Physiological Reviews 96:3, pages 1093-1126.
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J G Choi, Y Dang, S Abraham, H Ma, J Zhang, H Guo, Y Cai, J G Mikkelsen, H Wu, P Shankar & N Manjunath. (2016) Lentivirus pre-packed with Cas9 protein for safer gene editing. Gene Therapy 23:7, pages 627-633.
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C A Giménez, M Ielpi, A Mutto, L Grosembacher, P Argibay & F Pereyra-Bonnet. (2016) CRISPR-on system for the activation of the endogenous human INS gene. Gene Therapy 23:6, pages 543-547.
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Joshua Hunsberger, Josh Neubert, Jason A. Wertheim, Julie Allickson & Anthony Atala. (2016) Bioengineering Priorities on a Path to Ending Organ Shortage. Current Stem Cell Reports 2:2, pages 118-127.
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Lauren R. Polstein, Pablo Perez-Pinera, D. Dewran Kocak, Christopher M. Vockley, Peggy Bledsoe, Lingyun Song, Alexias Safi, Gregory E. Crawford, Timothy E. Reddy & Charles A. Gersbach. (2015) Genome-wide specificity of DNA binding, gene regulation, and chromatin remodeling by TALE- and CRISPR/Cas9-based transcriptional activators. Genome Research 25:8, pages 1158-1169.
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Monique G.P. van der Wijst, Christian Huisman, Archibold Mposhi, Gerard Roelfes & Marianne G. Rots. (2015) Targeting Nrf2 in healthy and malignant ovarian epithelial cells: Protection versus promotion. Molecular Oncology 9:7, pages 1259-1273.
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Ami M. Kabadi, Pratiksha I. Thakore, Christopher M. Vockley, David G. Ousterout, Tyler M. Gibson, Farshid Guilak, Timothy E. Reddy & Charles A. Gersbach. (2014) Enhanced MyoD-Induced Transdifferentiation to a Myogenic Lineage by Fusion to a Potent Transactivation Domain. ACS Synthetic Biology 4:6, pages 689-699.
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Alexandre Juillerat, Coline Pessereau, Gwendoline Dubois, Valérie Guyot, Alan Maréchal, Julien Valton, Fayza Daboussi, Laurent Poirot, Aymeric Duclert & Philippe Duchateau. (2015) Optimized tuning of TALEN specificity using non-conventional RVDs. Scientific Reports 5:1.
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Ami M. Kabadi, David G. Ousterout, Isaac B. Hilton & Charles A. Gersbach. (2014) Multiplex CRISPR/Cas9-based genome engineering from a single lentiviral vector. Nucleic Acids Research 42:19, pages e147-e147.
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