Charles Gersbach

Research Themes

Biomaterials, Drug & Gene Delivery, Immune Engineering, Synthetic & Systems Biology, Tissue Engineering & Regenerative Medicine

Research Interests

Gene therapy, genomics and epigenomics, biomolecular and cellular engineering, regenerative medicine, and synthetic biology.

Education

• B.S. Georgia Institute of Technology, 2001
• Ph.D. Georgia Institute of Technology, 2006

Positions

• John W. Strohbehn Distinguished Professor of Biomedical Engineering
• Professor of Biomedical Engineering
• Associate Professor of Surgery
• Associate Professor in Cell Biology
• Associate Professor in Orthopaedic Surgery
• Affiliate of the Duke Regeneration Center
• Core Faculty in Innovation & Entrepreneurship
• Associate of the Duke Initiative for Science & Society
• Member of the Duke Cancer Institute

Awards, Honors, and Distinctions

• Fellow (NAI). National Academy of Inventors. 2022
• John W. Strohbehn Distinguished Professor of Biomedical Engineering . Duke University. 2021
• Stansell Family Distinguished Research Award . Pratt School of Engineering. 2019
• Allen Distinguished Investigator. Paul G. Allen Frontiers Group. 2017
• Fellow. American Institute for Medical and Biological Engineering. 2017
• Thomas Langford Lectureship Award. Duke University. 2016
• Outstanding New Investigator. American Society of Gene and Cell Therapy. 2014
• Capers and Marion McDonald Teaching and Research Award. Duke University. 2013
• Faculty Early Career Development (CAREER) Program. National Science Foundation. 2012
• Rising Star Award. Society for Physical Regulation in Biology and Medicine. 2012
• Basil Ou2019Connor Starter Scholar Research Award. March of Dimes Foundation. 2011
• Ralph E. Powe Junior Faculty Enhancement Award. Oak Ridge Associated Universities. 2011
• NIH Directoru2019s New Innovator Award. National Institutes of Health. 2011
• Hartwell Individual Biomedical Research Award. The Hartwell Foundation. 2010

Courses Taught

• PHARM 494: Research Independent Study
• PHARM 493: Research Independent Study
• EGR 491: Projects in Engineering
• EGR 393: Research Projects in Engineering
• CELLBIO 493: Research Independent Study
• BME 791: Graduate Independent Study
• BME 590L: Special Topics with Lab
• BME 564L: Genome Engineering Lab (GE, MC)
• BME 562: Biology by Design (GE, MC)
• BME 494: Projects in Biomedical Engineering (GE)
• BME 493: Projects in Biomedical Engineering (GE)
• BME 493-1: Projects in Biomedical Engineering (GE)
• BME 394: Projects in Biomedical Engineering (GE)

Publications

• Liu S, Hamilton MC, Cowart T, Barrera A, Bounds LR, Nelson AC, et al. Characterization and bioinformatic filtering of ambient gRNAs in single-cell CRISPR screens using CLEANSER. bioRxiv. 2024 Sep 4;
• Gao Y, Shonai D, Trn M, Zhao J, Soderblom EJ, Garcia-Moreno SA, et al. Proximity analysis of native proteomes reveals phenotypic modifiers in a mouse model of autism and related neurodevelopmental conditions. Nat Commun. 2024 Aug 9;15(1):6801.
• Roger AL, Biswas DD, Huston ML, Le D, Bailey AM, Pucci LA, et al. Respiratory characterization of a humanized Duchenne muscular dystrophy mouse model. Respir Physiol Neurobiol. 2024 Aug;326:104282.
• McCutcheon SR, Rohm D, Iglesias N, Gersbach CA. Epigenome editing technologies for discovery and medicine. Nature biotechnology. 2024 Aug;42(8):1199–217.
• Mu W, Luo T, Barrera A, Bounds LR, Klann TS, Ter Weele M, et al. Machine learning methods for predicting guide RNA effects in CRISPR epigenome editing experiments. bioRxiv. 2024 Apr 19;
• Yao D, Tycko J, Oh JW, Bounds LR, Gosai SJ, Lataniotis L, et al. Multicenter integrated analysis of noncoding CRISPRi screens. Nat Methods. 2024 Apr;21(4):723–34.
• Barzi M, Chen T, Gonzalez TJ, Pankowicz FP, Oh SH, Streff HL, et al. A humanized mouse model for adeno-associated viral gene therapy. Nat Commun. 2024 Mar 4;15(1):1955.
• Kabadi AM, Mejia-Guerra MK, Graef JD, Khan SZ, Walton EM, Wang X, et al. AAV-based CRISPR-Cas9 genome editing: Challenges and engineering opportunities. Current Opinion in Biomedical Engineering. 2024 Mar 1;29.
• Cosgrove BD, Bounds LR, Taylor CK, Su AL, Rizzo AJ, Barrera A, et al. Mechanosensitive genomic enhancers potentiate the cellular response to matrix stiffness. bioRxiv. 2024 Jan 10;
• Lee JH, Shores KL, Breithaupt JJ, Lee CS, Fodera DM, Kwon JB, et al. PCSK9 activation promotes early atherosclerosis in a vascular microphysiological system. APL bioengineering. 2023 Dec;7(4):046103.
• McCutcheon SR, Swartz AM, Brown MC, Barrera A, McRoberts Amador C, Siklenka K, et al. Transcriptional and epigenetic regulators of human CD8+ T cell function identified through orthogonal CRISPR screens. Nat Genet. 2023 Dec;55(12):2211–23.
• Fletcher RB, Stokes LD, Kelly IB, Henderson KM, Vallecillo-Viejo IC, Colazo JM, et al. Nonviral In Vivo Delivery of CRISPR-Cas9 Using Protein-Agnostic, High-Loading Porous Silicon and Polymer Nanoparticles. ACS nano. 2023 Sep;17(17):16412–31.
• McCutcheon SR, Swartz AM, Brown MC, Barrera A, Amador CM, Siklenka K, et al. Orthogonal CRISPR screens to identify transcriptional and epigenetic regulators of human CD8 T cell function. bioRxiv. 2023 May 1;
• Kwon JB, Knapp A, Hill A, Browoleit KJ, An S, Sundseth S, et al. Transient Delivery of Epigenome Editors Stably Represses PCSK9 and Lowers LDL Cholesterol in Non-Human Primates. In: MOLECULAR THERAPY. 2023. p. 3–3.
• Tarantal AF, Martinez M, Sanz L, Lee C, O’Geen H, Asokan A, et al. AAV Serotype Tropism and Editing in Young Rhesus Monkeys. In: MOLECULAR THERAPY. 2023. p. 42–42.
• Sitton MJ, Khodabukus A, Bohning JD, Bursac N, Gersbach CA. Modeling Gene Editing Outcomes in Microphysiological Human Tissue System Models of Duchenne Muscular Dystrophy. In: MOLECULAR THERAPY. 2023. p. 65–65.
• Yan R, Cigliola V, Oonk KA, Petrover Z, DeLuca S, Wolfson DW, et al. An enhancer-based gene-therapy strategy for spatiotemporal control of cargoes during tissue repair. Cell stem cell. 2023 Jan;30(1):96-111.e6.
• Gonzalez TJ, Simon KE, Blondel LO, Fanous MM, Roger AL, Maysonet MS, et al. Cross-species evolution of a highly potent AAV variant for therapeutic gene transfer and genome editing. Nat Commun. 2022 Oct 10;13(1):5947.
• Beyersdorf JP, Bawage S, Iglesias N, Peck HE, Hobbs RA, Wroe JA, et al. Robust, Durable Gene Activation In Vivo via mRNA-Encoded Activators. ACS nano. 2022 Apr;16(4):5660–71.
• Gough V, Engstrom C, Mejia-Guerra K, Bohning J, Liu S, Majoros W, et al. A High-Throughput Screen for CRISPR-Cas9-Mediated Exon Deletion. In: MOLECULAR THERAPY. 2022. p. 456–456.
• McCutcheon S, Amador CM, Barrera A, Humayun L, Gersbach C. CRISPR-Based Epigenome Editing Screens in Primary Human T Cells. In: MOLECULAR THERAPY. 2022. p. 165–6.
• Walton EM, Black JB, Castillo L, Gersbach CA, Asokan A. Host Transcription Factors and Co-Opted Signaling Pathways Orchestrate Epigenetic Modulation of the AAV Vector Genome. In: MOLECULAR THERAPY. 2022. p. 430–430.
• Butterfield G, Rohm D, Roberts A, Nethery M, Rizzo A, Barrangou R, et al. A Novel Cas9 System with Robust Genome and Epigenome Editing in Human Cells. In: MOLECULAR THERAPY. 2022. p. 271–2.
• Sitton MJ, Khodabukus A, McCullough KT, Bursac N, Gersbach CA. CRISPR Genome Editing in Microphysiological Human Tissue System Models of Duchenne Muscular Dystrophy. In: MOLECULAR THERAPY. 2022. p. 500–500.
• Gonzalez TJ, Blondel L, Rosales A, Daniels H, Gersbach CA, Asokan A. Enhanced CRISPR/Cas9 Genome Editing in Heart and Skeletal Muscle with a Potent New AAV Variant. In: MOLECULAR THERAPY. 2022. p. 211–2.
• Pickar-Oliver A, Gough V, Bohning JD, Liu S, Robinson-Hamm JN, Daniels H, et al. Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy. Mol Ther. 2021 Nov 3;29(11):3243–57.
• Hakim CH, Kumar SRP, Pérez-López DO, Wasala NB, Zhang D, Yue Y, et al. Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models. Nature communications. 2021 Nov;12(1):6769.
• Wang L, Wang E, Prado Balcazar J, Wu Z, Xiang K, Wang Y, et al. Chromatin Remodeling of Colorectal Cancer Liver Metastasis is Mediated by an HGF-PU.1-DPP4 Axis. Adv Sci (Weinh). 2021 Oct;8(19):e2004673.
• Gemberling MP, Siklenka K, Rodriguez E, Tonn-Eisinger KR, Barrera A, Liu F, et al. Transgenic mice for in vivo epigenome editing with CRISPR-based systems. Nat Methods. 2021 Aug;18(8):965–74.
• Abdeen AA, Cosgrove BD, Gersbach CA, Saha K. Integrating Biomaterials and Genome Editing Approaches to Advance Biomedical Science. Vol. 23. 2021.
• McCall A, Bailey A, Pucci L, Dhindsa J, Robinson‐Hamm J, Gersbach C, et al. Respiratory Pathology in a Humanized Mouse Model of Duchenne Muscular Dystrophy. In: The FASEB Journal. Wiley; 2021.
• Saha K, Sontheimer EJ, Brooks PJ, Dwinell MR, Gersbach CA, Liu DR, et al. The NIH Somatic Cell Genome Editing program. Nature. 2021 Apr;592(7853):195–204.
• Bodle JC, Gersbach CA. CRISPR Clocks: The Times They Are a-Changin'. The CRISPR journal. 2021 Apr;4(2):160–3.
• Seo J, Koçak DD, Bartelt LC, Williams CA, Barrera A, Gersbach CA, et al. AP-1 subunits converge promiscuously at enhancers to potentiate transcription. Genome Res. 2021 Apr;31(4):538–50.
• Walejko JM, Christopher BA, Crown SB, Zhang G-F, Pickar-Oliver A, Yoneshiro T, et al. Branched-chain α-ketoacids are preferentially reaminated and activate protein synthesis in the heart. Nat Commun. 2021 Mar 15;12(1):1680.
• Pickar-Oliver A, Nelson C, Bohning J, Gough V, Perelli RM, Landstrom AP, et al. Development of AAV-Based CRISPR/Cas9 Therapies for Correcting Duchenne Muscular Dystrophy by Targeted Genomic Integration. In: MOLECULAR THERAPY. 2021. p. 163–4.
• Walton EM, Black JB, Gersbach CA, Asokan A. A CRISPRa Screen Identifies Transcription Factors That Can Silence or Activate rAAV Genomes. In: MOLECULAR THERAPY. 2021. p. 150–1.
• Kwon JB, Ettyreddy AR, Vankara A, Bohning JD, Devlin G, Hauschka SD, et al. In Vivo Gene Editing of Muscle Stem Cells with Adeno-Associated Viral Vectors in a Mouse Model of Duchenne Muscular Dystrophy. Mol Ther Methods Clin Dev. 2020 Dec 11;19:320–9.
• Black JB, McCutcheon SR, Dube S, Barrera A, Klann TS, Rice GA, et al. Master Regulators and Cofactors of Human Neuronal Cell Fate Specification Identified by CRISPR Gene Activation Screens. Cell Rep. 2020 Dec 1;33(9):108460.
• Bulaklak K, Gersbach CA. The once and future gene therapy. Nature communications. 2020 Nov;11(1):5820.
• Carullo NVN, Phillips Iii RA, Simon RC, Soto SAR, Hinds JE, Salisbury AJ, et al. Enhancer RNAs predict enhancer-gene regulatory links and are critical for enhancer function in neuronal systems. Nucleic acids research. 2020 Sep;48(17):9550–70.
• Mao M, Chang C-C, Pickar-Oliver A, Cervia LD, Wang L, Ji J, et al. Redirecting Vesicular Transport to Improve Nonviral Delivery of Molecular Cargo. Adv Biosyst. 2020 Aug;4(8):e2000059.
• Cosgrove BD, Gersbach CA. Unwinding the Role of FACT in Cas9-based Genome Editing. Molecular cell. 2020 Aug;79(3):365–7.
• Gough V, Gersbach CA. Immunity to Cas9 as an Obstacle to Persistent Genome Editing. Molecular therapy : the journal of the American Society of Gene Therapy. 2020 Jun;28(6):1389–91.
• Kwon JB, Vankara A, Ettyreddy AR, Bohning JD, Gersbach CA. Myogenic Progenitor Cell Lineage Specification by CRISPR/Cas9-Based Transcriptional Activators. Stem cell reports. 2020 May;14(5):755–69.
• Dicks A, Wu C-L, Steward N, Adkar SS, Gersbach CA, Guilak F. Prospective isolation of chondroprogenitors from human iPSCs based on cell surface markers identified using a CRISPR-Cas9-generated reporter. Stem cell research & therapy. 2020 Feb;11(1):66.
• Oliver A, Nelson C, Bohning J, Gilcrest K, Gersbach C. Development of AAV-Based CRISPR/Cas9 Therapies for Correcting Duchenne Muscular Dystrophy by Targeted Genomic Integration. In: MOLECULAR THERAPY. 2020. p. 6–6.
• Kwon J, Vankara A, Bohning J, Delvin G, Asokan A, Gersbach C. Targeting Muscle Satellite Cells for In Vivo Gene Editing With Adeno-Associated Viral Vectors for Duchenne Muscular Dystrophy. In: MOLECULAR THERAPY. 2020. p. 330–330.
• Sitton MJ, Majoros WH, Kirsch DG, Van Mater DS, Gersbach CA. Targeted Genomic Integration to Restore the NF1 Coding Sequence in Models of Neurofibromatosis Type I (NF1). In: MOLECULAR THERAPY. 2020. p. 278–278.
• Gonzalez TJ, Havlik LP, Fanous M, Simon K, Edwards L, Kwon J, et al. Cross-Species Evolution of Synthetic AAV Strains for Clinical Translation. In: MOLECULAR THERAPY. 2020. p. 12–3.
• McCall A, Bailey A, Pucci L, Dhindsa J, Strickland L, Robinson-Hamm J, et al. Respiratory Dysfunction in a novel mouse model of Duchenne Muscular Dystrophy. In: FASEB JOURNAL. 2020.
• Bulaklak K, Bohning J, Gough V, Daniels H, McCall A, ElMallah M, et al. CRISPR-Mediated Gene Correction in a Severe Humanized Mouse Model of Duchenne Muscular Dystrophy. In: MOLECULAR THERAPY. 2020. p. 218–9.
• Nelson CE, Duvall CL, Prokop A, Gersbach CA, Davidson JM. Gene delivery into cells and tissues. In: Principles of Tissue Engineering. 2020. p. 519–54.
• Pickar-Oliver A, Black JB, Lewis MM, Mutchnick KJ, Klann TS, Gilcrest KA, et al. Targeted transcriptional modulation with type I CRISPR-Cas systems in human cells. In: Nat Biotechnol. 2019. p. 1493–501.
• Huang J, Chen M, Xu ES, Luo L, Ma Y, Huang W, et al. Genome-wide CRISPR Screen to Identify Genes that Suppress Transformation in the Presence of Endogenous KrasG12D. Sci Rep. 2019 Nov 20;9(1):17220.
• Evans BC, Fletcher RB, Kilchrist KV, Dailing EA, Mukalel AJ, Colazo JM, et al. An anionic, endosome-escaping polymer to potentiate intracellular delivery of cationic peptides, biomacromolecules, and nanoparticles. Nature communications. 2019 Nov;10(1):5012.
• Nance ME, Shi R, Hakim CH, Wasala NB, Yue Y, Pan X, et al. AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice. Molecular therapy : the journal of the American Society of Gene Therapy. 2019 Sep;27(9):1568–85.
• Kwon JB, Gersbach CA. Jumping at the chance for precise DNA integration. Nature biotechnology. 2019 Sep;37(9):1004–6.
• Pickar-Oliver A, Gersbach CA. The next generation of CRISPR-Cas technologies and applications. Nature reviews Molecular cell biology. 2019 Aug;20(8):490–507.
• Kocak DD, Josephs EA, Bhandarkar V, Adkar SS, Kwon JB, Gersbach CA. Increasing the specificity of CRISPR systems with engineered RNA secondary structures. Nature biotechnology. 2019 Jun;37(6):657–66.
• Kwon J, Vankara A, Gersbach C. Directing Skeletal Myogenic Progenitor Cell Lineage Specification with CRISPR/Cas9-Based Transcriptional Activators. In: MOLECULAR THERAPY. CELL PRESS; 2019. p. 454–5.
• Pickar-Oliver A, Nelson C, Bohning J, Gemberling M, Bulaklak K, Gersbach CA. Development of AAV-Based CRISPR/Cas9 Therapies for Correcting Duchenne Muscular Dystrophy by Targeted Genomic Integration. In: MOLECULAR THERAPY. CELL PRESS; 2019. p. 180–1.
• Nelson C, Wu Y, Gemberling M, Oliver M, Bohning JD, Robinson-Hamm JN, et al. Long-Term Evaluation of AAV-CRISPR Genome Editing for Duchenne Muscular Dystrophy. In: MOLECULAR THERAPY. CELL PRESS; 2019. p. 46–7.
• Bulaklak K, Robinson-Hamm J, Gough V, Nelson CE, Madigan V, Asokan A, et al. AAV-Mediated Deletion of a Large Mutational Hotspot for Treatment of Duchenne Muscular Dystrophy. In: MOLECULAR THERAPY. CELL PRESS; 2019. p. 377–377.
• Nelson CE, Wu Y, Gemberling MP, Oliver ML, Waller MA, Bohning JD, et al. Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy. Nat Med. 2019 Mar;25(3):427–32.
• Chen L-F, Lin YT, Gallegos DA, Hazlett MF, Gómez-Schiavon M, Yang MG, et al. Enhancer Histone Acetylation Modulates Transcriptional Bursting Dynamics of Neuronal Activity-Inducible Genes. Cell Rep. 2019 Jan 29;26(5):1174-1188.e5.
• Nelson CE, Gersbach CA. Genome Editing for Duchenne Muscular Dystrophy. In: Muscle Gene Therapy, Second Edition. 2019. p. 383–403.
• Adkar SS, Wu C-L, Willard VP, Dicks A, Ettyreddy A, Steward N, et al. Step-Wise Chondrogenesis of Human Induced Pluripotent Stem Cells and Purification Via a Reporter Allele Generated by CRISPR-Cas9 Genome Editing. Stem cells (Dayton, Ohio). 2019 Jan;37(1):65–76.
• Breithaupt JJ, Kwon J, Ettyreddy A, Zhang X, Gersbach C, Truskey GA. PCSK9 Knockdown Decreases Inflammatory Markers and U937 Monocyte Accumulation in a Tissue-Engineered Blood Vessel Model. In: CIRCULATION. 2019.
• Hakim CH, Wasala NB, Nelson CE, Wasala LP, Yue Y, Louderman JA, et al. AAV CRISPR editing rescues cardiac and muscle function for 18 months in dystrophic mice. JCI insight. 2018 Dec;3(23):124297.
• Gersbach C, Nelson C, Robinson-Hamm J, Kwon J, Gough V, Gemberling M. NEW THERAPEUTIC APPROACHES AND THEIR READOUT. In: Neuromuscular Disorders. Elsevier BV; 2018. p. S90–S90.
• Huynh NPT, Brunger JM, Gloss CC, Moutos FT, Gersbach CA, Guilak F. Genetic Engineering of Mesenchymal Stem Cells for Differential Matrix Deposition on 3D Woven Scaffolds. Tissue engineering Part A. 2018 Oct;24(19–20):1531–44.
• Black JB, Gersbach CA. Synthetic transcription factors for cell fate reprogramming. Current opinion in genetics & development. 2018 Oct;52:13–21.
• McDowell IC, Barrera A, D’Ippolito AM, Vockley CM, Hong LK, Leichter SM, et al. Glucocorticoid receptor recruits to enhancers and drives activation by motif-directed binding. Genome Res. 2018 Sep;28(9):1272–84.
• Gersbach CA. Gene delivery and biomedical engineering. Current Opinion in Biomedical Engineering. 2018 Sep 1;7:iii–v.
• D’Ippolito AM, McDowell IC, Barrera A, Hong LK, Leichter SM, Bartelt LC, et al. Pre-established Chromatin Interactions Mediate the Genomic Response to Glucocorticoids. Cell Syst. 2018 Aug 22;7(2):146-160.e7.
• Gersbach C, Nelson C, Robinson-Hamm J, Kwon J. Genome editing as a therapy for Duchenne muscular dystrophy. In: ABSTRACTS OF PAPERS OF THE AMERICAN CHEMICAL SOCIETY. AMER CHEMICAL SOC; 2018.
• Holtzman L, Gersbach CA. Editing the Epigenome: Reshaping the Genomic Landscape. Vol. 19. 2018.
• Klann TS, Black JB, Gersbach CA. CRISPR-based methods for high-throughput annotation of regulatory DNA. Current opinion in biotechnology. 2018 Aug;52:32–41.
• Kwon J, Gersbach C. Targeting Muscle Satellite Cells with Adeno-Associated Viral Vectors. In: MOLECULAR THERAPY. CELL PRESS; 2018. p. 438–438.
• Adkar SS, Wu C-L, Willard VP, Dicks A, Ettyreddy A, Steward N, et al. Highly Efficient Chondrogenic Differentiation of Human iPSCs and Purification via a Reporter Allele Generated by CRISPR-Cas9 Genome Editing. In: MOLECULAR THERAPY. CELL PRESS; 2018. p. 36–36.
• Hakim CH, Wasala N, Nelson C, Wasala L, Yue Y, Zhang K, et al. Optimization of the gRNA Vector Dose Enhances Long-Term Systemic AAV CRISPR Therapy in Muscular Dystrophy. In: MOLECULAR THERAPY. CELL PRESS; 2018. p. 370–370.
• Nance ME, Shi R, Hakim CH, Wasala N, Yue Y, Pan X, et al. AAV-9 CRISPR Mediated Satellite Cell Editing Restored Dystrophin Expression after Complete Degeneration in a Whole Muscle Graft Model. In: MOLECULAR THERAPY. CELL PRESS; 2018. p. 393–393.
• Robinson-Hamm JN, Nelson CE, Gemberling M, Gough V, Rivera RMC, Aartsma-Rus A, et al. Dystrophin Restoration in a Humanized Mouse Model of Duchenne Muscular Dystrophy by Gene Editing with S. Aureus Cas9. In: MOLECULAR THERAPY. CELL PRESS; 2018. p. 118–9.
• Nelson C, Gemberling M, Oliver ML, Hakim CH, Rivera RMC, Asokan A, et al. Long-Term Evaluation of Genome Editing Outcomes for Duchenne Muscular Dystrophy. In: MOLECULAR THERAPY. CELL PRESS; 2018. p. 431–2.
• Kocak DD, Gersbach CA. From CRISPR scissors to virus sensors. Nature. 2018 May;557(7704):168–9.
• Thakore PI, Kwon JB, Nelson CE, Rouse DC, Gemberling MP, Oliver ML, et al. RNA-guided transcriptional silencing in vivo with S. aureus CRISPR-Cas9 repressors. Nature communications. 2018 Apr;9(1):1674.
• Gersbach CA, Barrangou R. Pulling the genome in opposite directions to dissect gene networks. Genome biology. 2018 Mar;19(1):42.
• Gemberling M, Gersbach CA. Boosting, Not Breaking: CRISPR Activators Treat Disease Models. Molecular therapy : the journal of the American Society of Gene Therapy. 2018 Feb;26(2):334–6.
• Pickar AK, Gersbach CA. Gene therapies for hemophilia hit the mark in clinical trials. Nature medicine. 2018 Feb;24(2):121–2.
• Huang J, Chen M, Whitley MJ, Kuo H-C, Xu ES, Walens A, et al. Generation and comparison of CRISPR/Cas9 and Cre-mediated genetically engineered mouse models of sarcoma. In: CLINICAL CANCER RESEARCH. AMER ASSOC CANCER RESEARCH; 2018. p. 44–5.
• Klann TS, Crawford GE, Reddy TE, Gersbach CA. Screening Regulatory Element Function with CRISPR/Cas9-based Epigenome Editing. Methods Mol Biol. 2018;1767:447–80.
• Gersbach CA. Editorial Overview: Synthetic biology and biomedical engineering. Current Opinion in Biomedical Engineering. 2017 Dec 1;4:vi–vii.
• Manandhar D, Song L, Kabadi A, Kwon JB, Edsall LE, Ehrlich M, et al. Incomplete MyoD-induced transdifferentiation is associated with chromatin remodeling deficiencies. Nucleic Acids Res. 2017 Nov 16;45(20):11684–99.
• Pathak GP, Spiltoir JI, Höglund C, Polstein LR, Heine-Koskinen S, Gersbach CA, et al. Bidirectional approaches for optogenetic regulation of gene expression in mammalian cells using Arabidopsis cryptochrome 2. Nucleic acids research. 2017 Nov;45(20):e167.
• Nelson CE, Robinson-Hamm JN, Gersbach CA. Genome engineering: a new approach to gene therapy for neuromuscular disorders. Nature reviews Neurology. 2017 Nov;13(11):647–61.
• Polstein LR, Juhas M, Hanna G, Bursac N, Gersbach CA. An Engineered Optogenetic Switch for Spatiotemporal Control of Gene Expression, Cell Differentiation, and Tissue Morphogenesis. ACS synthetic biology. 2017 Nov;6(11):2003–13.
• Adkar SS, Brunger JM, Willard VP, Wu C-L, Gersbach CA, Guilak F. Genome Engineering for Personalized Arthritis Therapeutics. Trends in molecular medicine. 2017 Oct;23(10):917–31.
• Farhang N, Brunger JM, Stover JD, Thakore PI, Lawrence B, Guilak F, et al. ^* CRISPR-Based Epigenome Editing of Cytokine Receptors for the Promotion of Cell Survival and Tissue Deposition in Inflammatory Environments. Tissue engineering Part A. 2017 Aug;23(15–16):738–49.
• Huang J, Chen M, Whitley MJ, Kuo H-C, Xu ES, Walens A, et al. Generation and comparison of CRISPR-Cas9 and Cre-mediated genetically engineered mouse models of sarcoma. Nat Commun. 2017 Jul 10;8:15999.
• Huang J, Chen M, Whitley MJ, Kuo H-C, Walens A, Mowery YM, et al. Abstract 2810: Using CRISPR/Cas9 to generate primary soft tissue sarcoma in genetically engineered and wild-type mice. In: Cancer Research. American Association for Cancer Research (AACR); 2017. p. 2810–2810.
• Black JB, Perez-Pinera P, Gersbach CA. Mammalian Synthetic Biology: Engineering Biological Systems. Annual review of biomedical engineering. 2017 Jun;19:249–77.
• Klann TS, Black JB, Chellappan M, Safi A, Song L, Hilton IB, et al. CRISPR-Cas9 epigenome editing enables high-throughput screening for functional regulatory elements in the human genome. Nat Biotechnol. 2017 Jun;35(6):561–8.
• Thakore PI, Kwon JB, Nelson CE, Gemberling MP, Oliver M, Gersbach CA. In Vivo Gene Silencing with S-aureus CRISPR-Cas9 Repressors. In: MOLECULAR THERAPY. CELL PRESS; 2017. p. 230–230.
• Robinson-Hamm JN, Nelson CE, Gemberling M, Rivera RMC, Aartsma-Rus A, Asokan A, et al. Dystrophin Restoration in a Humanized Mouse Model of Duchenne Muscular Dystrophy by Gene Editing with S. aureus Cas9. In: MOLECULAR THERAPY. CELL PRESS; 2017. p. 17–17.
• Nelson C, Gemberling M, Madhavan S, Rivera RMC, Asokan A, Gersbach CA. Long-Term Characterization of In Vivo Genome Editing in a Mouse Model of Duchenne Muscular Dystrophy. In: MOLECULAR THERAPY. CELL PRESS; 2017. p. 100–1.
• Brunger JM, Zutshi A, Willard VP, Gersbach CA, Guilak F. CRISPR/Cas9 Editing of Murine Induced Pluripotent Stem Cells for Engineering Inflammation-Resistant Tissues. Arthritis & rheumatology (Hoboken, NJ). 2017 May;69(5):1111–21.
• Brunger JM, Zutshi A, Willard VP, Gersbach CA, Guilak F. Genome Engineering of Stem Cells for Autonomously Regulated, Closed-Loop Delivery of Biologic Drugs. Stem cell reports. 2017 May;8(5):1202–13.
• Barrangou R, Gersbach CA. Expanding the CRISPR Toolbox: Targeting RNA with Cas13b. Molecular cell. 2017 Feb;65(4):582–4.
• Hilton IB, Gersbach CA. Genetic engineering: Chemical control for CRISPR editing. Nature chemical biology. 2017 Jan;13(1):2–3.
• Housden BE, Muhar M, Gemberling M, Gersbach CA, Stainier DYR, Seydoux G, et al. Loss-of-function genetic tools for animal models: cross-species and cross-platform differences. Nature reviews Genetics. 2017 Jan;18(1):24–40.
• Gersbach CA, Nelson CE, Robinson-Hamm JN. Genome editing for Duchenne muscular dystrophy. In: HUMAN GENE THERAPY. MARY ANN LIEBERT, INC; 2016. p. A20–A20.
• Lee J, Xu L, Gibson TM, Gersbach CA, Sullenger BA. Differential effects of toll-like receptor stimulation on mRNA-driven myogenic conversion of human and mouse fibroblasts. Biochem Biophys Res Commun. 2016 Sep 23;478(3):1484–90.
• Black JB, Adler AF, Wang H-G, D’Ippolito AM, Hutchinson HA, Reddy TE, et al. Targeted Epigenetic Remodeling of Endogenous Loci by CRISPR/Cas9-Based Transcriptional Activators Directly Converts Fibroblasts to Neuronal Cells. Cell Stem Cell. 2016 Sep 1;19(3):406–14.
• Robinson-Hamm JN, Gersbach CA. Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy. Human genetics. 2016 Sep;135(9):1029–40.
• Moutos FT, Glass KA, Compton SA, Ross AK, Gersbach CA, Guilak F, et al. Anatomically shaped tissue-engineered cartilage with tunable and inducible anticytokine delivery for biological joint resurfacing. Proceedings of the National Academy of Sciences of the United States of America. 2016 Aug;113(31):E4513–22.
• Hwang PY, Jing L, Chen J, Lim F-L, Tang R, Choi H, et al. N-cadherin is Key to Expression of the Nucleus Pulposus Cell Phenotype under Selective Substrate Culture Conditions. Scientific reports. 2016 Jun;6:28038.
• Nelson CE, Gersbach CA. Engineering Delivery Vehicles for Genome Editing. Annual review of chemical and biomolecular engineering. 2016 Jun;7:637–62.
• Adkar SS, Willard VP, Brunger JM, Shiao KT, Gersbach CA, Guilak F. 318. Targeted Genome Editing of Human Induced Pluripotent Stem Cells Using CRISPR/CAS9 to Generate a Knock-in Type II Collagen Reporter for the Purification of Chondrogenic Cells. In: Molecular Therapy. Elsevier BV; 2016. p. S128–S128.
• Kabadi AM, Klann TS, Chellappan M, Kwon J, Reddy TE, Gersbach CA. 525. Directed Molecular Evolution of Transcription Factors in Mammalian Cells for Enhanced Directed Cell Differentiation. In: Molecular Therapy. Elsevier BV; 2016. p. S210–S210.
• Landau DJ, Brooks ED, Perez-Pinera PP, Amarasekara H, Mefferd A, Li S, et al. 311. In Vivo Zinc Finger Nuclease-Mediated Targeted Integration of a Glucose-6-Phosphatase Transgene Enhances Biochemical Correction in Mice with Glycogen Storage Disease Type IA. In: Molecular Therapy. Elsevier BV; 2016. p. S125–S125.
• Nelson C, Gemberling M, Hakim CH, Ousterout DG, Thakore PI, Castellanos RM, et al. 482. Local and Systemic Gene Editing in a Mouse Model of Duchenne Muscular Dystrophy. In: Molecular Therapy. Elsevier BV; 2016. p. S191–S191.
• Robinson-Hamm JN, Nelson CE, Rivera RMC, Aartsma-Rus A, Asokan A, Gersbach CA. 504. Restoration of Dystrophin Expression by Gene Editing with S. aureus Cas9 in Models of Duchenne Muscular Dystrophy. In: Molecular Therapy. Elsevier BV; 2016. p. S201–S201.
• Glass KA, Rowland CR, Ettyreddy AR, Brunger JM, Huynh NPT, Gersbach CA, et al. 502. Biomaterial-Mediated Lentiviral Delivery of Anti-Inflammatory Genes in Cartilage-Derived Matrix Hemispheres. In: Molecular Therapy. Elsevier BV; 2016. p. S199–200.
• Landau DJ, Brooks ED, Perez-Pinera P, Amarasekara H, Mefferd A, Li S, et al. In Vivo Zinc Finger Nuclease-mediated Targeted Integration of a Glucose-6-phosphatase Transgene Promotes Survival in Mice With Glycogen Storage Disease Type IA. Mol Ther. 2016 Apr;24(4):697–706.
• Josephs EA, Kocak DD, Fitzgibbon CJ, McMenemy J, Gersbach CA, Marszalek PE. Structure and specificity of the RNA-guided endonuclease Cas9 during DNA interrogation, target binding and cleavage. Nucleic acids research. 2016 Mar;44(5):2474.
• Nelson CE, Gersbach CA. Cas9 loosens its grip on off-target sites. Nature biotechnology. 2016 Mar;34(3):298–9.
• Maeder ML, Gersbach CA. Genome-editing Technologies for Gene and Cell Therapy. Molecular therapy : the journal of the American Society of Gene Therapy. 2016 Mar;24(3):430–46.
• Josephs EA, Kocak DD, Fitzgibbon CJ, McMenemy J, Gersbach CA, Marszalek PE. A Molecule-Scale View of the Structure and Specificity of the RNA-Guided Endonuclease Cas9. In: Biophysical Journal. Elsevier BV; 2016. p. 238a-238a.
• Thakore PI, Black JB, Hilton IB, Gersbach CA. Editing the epigenome: technologies for programmable transcription and epigenetic modulation. Nature methods. 2016 Feb;13(2):127–37.
• Nelson CE, Hakim CH, Ousterout DG, Thakore PI, Moreb EA, Castellanos Rivera RM, et al. In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy. Science. 2016 Jan 22;351(6271):403–7.
• Ousterout DG, Gersbach CA. Genome Editing for Neuromuscular Diseases. In: Advances in Experimental Medicine and Biology. 2016. p. 51–79.
• Ousterout DG, Gersbach CA. The Development of TALE Nucleases for Biotechnology. Methods in molecular biology (Clifton, NJ). 2016 Jan;1338:27–42.
• Thakore PI, Gersbach CA. Design, Assembly, and Characterization of TALE-Based Transcriptional Activators and Repressors. Methods in molecular biology (Clifton, NJ). 2016 Jan;1338:71–88.
• Doudna JA, Gersbach CA. Genome editing: the end of the beginning. Genome biology. 2015 Dec;16:292.
• Thakore PI, D’Ippolito AM, Song L, Safi A, Shivakumar NK, Kabadi AM, et al. Highly specific epigenome editing by CRISPR-Cas9 repressors for silencing of distal regulatory elements. Nat Methods. 2015 Dec;12(12):1143–9.
• Hilton IB, Gersbach CA. Enabling functional genomics with genome engineering. Genome research. 2015 Oct;25(10):1442–55.
• Josephs EA, Kocak DD, Fitzgibbon CJ, McMenemy J, Gersbach CA, Marszalek PE. Structure and specificity of the RNA-guided endonuclease Cas9 during DNA interrogation, target binding and cleavage. Nucleic acids research. 2015 Oct;43(18):8924–41.
• Glass KA, Ross AK, Compton SA, Gersbach CA, Moutos FT, Estes BT, et al. Anatomically-Shaped Tissue-Engineered Cartilage with Tunable and Inducible Anti-Inflammatory Capabilities. In: TISSUE ENGINEERING PART A. MARY ANN LIEBERT, INC; 2015. p. S330–S330.
• Farhang N, Brunger JM, Stover JD, Thakore PI, Lawrence BD, Guilak F, et al. CRISPRi Immunomodulation for Tissue Engineering/Stem Cell Therapies Targeting Intervertebral Disc Degeneration. In: TISSUE ENGINEERING PART A. MARY ANN LIEBERT, INC; 2015. p. S170–S170.
• Polstein LR, Perez-Pinera P, Kocak DD, Vockley CM, Bledsoe P, Song L, et al. Genome-wide specificity of DNA binding, gene regulation, and chromatin remodeling by TALE- and CRISPR/Cas9-based transcriptional activators. Genome Res. 2015 Aug;25(8):1158–69.
• Kabadi AM, Thakore PI, Vockley CM, Ousterout DG, Gibson TM, Guilak F, et al. Enhanced MyoD-induced transdifferentiation to a myogenic lineage by fusion to a potent transactivation domain. ACS Synth Biol. 2015 Jun 19;4(6):689–99.
• Gibson TM, Gersbach CA. Single-molecule analysis of myocyte differentiation reveals bimodal lineage commitment. Integrative biology : quantitative biosciences from nano to macro. 2015 Jun;7(6):663–71.
• Hilton IB, D’Ippolito AM, Vockley CM, Thakore PI, Crawford GE, Reddy TE, et al. Epigenome editing by a CRISPR-Cas9-based acetyltransferase activates genes from promoters and enhancers. Nat Biotechnol. 2015 May;33(5):510–7.
• Brunger JM, Zutshi A, Willard VP, Gersbach CA, Guilak F. 504. Targeted Genome Engineering of Induced Pluripotent Stem Cells To Produce Auto-Regulated Inflammation Resistance for Musculoskeletal Regenerative Medicine. In: Molecular Therapy. Elsevier BV; 2015. p. S201–2.
• Nelson CE, Castellanos RM, Ann Ran F, Yan W, Zhang F, Asokan A, et al. 397. Correction of the Dystrophin Gene By the CRISPR/Cas9 System in a Mouse Model of Muscular Dystrophy. In: Molecular Therapy. Elsevier BV; 2015. p. S157–8.
• Thakore PI, D’Ippolito A, Song L, Safi A, Shivakumar NK, Kabadi AM, et al. 485. Targeted Epigenome Editing by CRISPR/Cas9-Based Repressors for Silencing of Distal Regulatory Elements. In: Molecular Therapy. Elsevier BV; 2015. p. S192–3.
• Frank CL, Liu F, Wijayatunge R, Song L, Biegler MT, Yang MG, et al. Regulation of chromatin accessibility and Zic binding at enhancers in the developing cerebellum. Nat Neurosci. 2015 May;18(5):647–56.
• Black J, Adler A, Hutchinson H, Wang H, Pitt G, Leong K, et al. 59. Multiplex Gene Activation by CRISPR/Cas9-Based Transcription Factors for the Direct Conversion of Fibroblasts to a Neuronal Phenotype. In: Molecular Therapy. Elsevier BV; 2015. p. S26–S26.
• Diekman BO, Thakore PI, O’Connor SK, Willard VP, Brunger JM, Christoforou N, et al. Knockdown of the cell cycle inhibitor p21 enhances cartilage formation by induced pluripotent stem cells. Tissue engineering Part A. 2015 Apr;21(7–8):1261–74.
• Ousterout DG, Kabadi AM, Thakore PI, Perez-Pinera P, Brown MT, Majoros WH, et al. Correction of dystrophin expression in cells from Duchenne muscular dystrophy patients through genomic excision of exon 51 by zinc finger nucleases. Molecular Therapy. 2015 Mar 5;23(3):523–32.
• Landau DJ, Brooks ED, Perez-Pinera P, Amarasekara H, Bird A, Li S, et al. Zinc finger nuclease-induced targeted integration of a glucose-6-phosphatase gene promotes survival in mice with glycogen storage disease type-IA. In: MOLECULAR GENETICS AND METABOLISM. ACADEMIC PRESS INC ELSEVIER SCIENCE; 2015. p. 316–316.
• Ousterout DG, Kabadi AM, Thakore PI, Perez-Pinera P, Brown MT, Majoros WH, et al. Correction of dystrophin expression in cells from Duchenne muscular dystrophy patients through genomic excision of exon 51 by zinc finger nucleases. Mol Ther. 2015 Mar;23(3):523–32.
• Polstein LR, Gersbach CA. A light-inducible CRISPR-Cas9 system for control of endogenous gene activation. Nature chemical biology. 2015 Mar;11(3):198–200.
• Ousterout DG, Kabadi AM, Thakore PI, Majoros WH, Reddy TE, Gersbach CA. Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy. Nat Commun. 2015 Feb 18;6:6244.
• Thakore PI, Gersbach CA. Genome Engineering for Therapeutic Applications. In: Translating Gene Therapy to the Clinic: Techniques and Approaches. 2015. p. 27–43.
• Polstein LR, Gersbach CA. A light-inducible CRISPR-Cas9 system for control of endogenous gene activation. Nature Chemical Biology. 2015 Jan 1;11(3):198–200.
• Chakraborty S, Ji H, Kabadi AM, Gersbach CA, Christoforou N, Leong KW. A CRISPR/Cas9-based system for reprogramming cell lineage specification. Stem cell reports. 2014 Dec;3(6):940–7.
• Chakraborty S, Ji H, Chen J, Gersbach CA, Leong KW. Vector modifications to eliminate transposase expression following piggyBac-mediated transgenesis. Scientific reports. 2014 Dec;4:7403.
• Kabadi AM, Gersbach CA. Special issue on engineered DNA-binding proteins. ACS synthetic biology. 2014 Oct;3(10):702–3.
• Gersbach CA. Genome engineering: the next genomic revolution. Nature methods. 2014 Oct;11(10):1009–11.
• Kabadi AM, Ousterout DG, Hilton IB, Gersbach CA. Multiplex CRISPR/Cas9-based genome engineering from a single lentiviral vector. Nucleic acids research. 2014 Oct;42(19):e147.
• Kabadi AM, Gersbach CA. Engineering synthetic TALE and CRISPR/Cas9 transcription factors for regulating gene expression. Methods (San Diego, Calif). 2014 Sep;69(2):188–97.
• Gersbach CA, Perez-Pinera P. Activating human genes with zinc finger proteins, transcription activator-like effectors and CRISPR/Cas9 for gene therapy and regenerative medicine. Expert opinion on therapeutic targets. 2014 Aug;18(8):835–9.
• Gersbach CA, Gaj T, Barbas CF. Synthetic zinc finger proteins: the advent of targeted gene regulation and genome modification technologies. Accounts of chemical research. 2014 Aug;47(8):2309–18.
• Glass KA, Link JM, Brunger JM, Moutos FT, Gersbach CA, Guilak F. Tissue-engineered cartilage with inducible and tunable immunomodulatory properties. Biomaterials. 2014 Jul;35(22):5921–31.
• Brunger JM, Huynh NPT, Moutos FT, Guilak F, Gersbach CA. Biomaterial-Mediated Lentiviral Gene Delivery for Osteochondral Tissue Engineering. In: MOLECULAR THERAPY. NATURE PUBLISHING GROUP; 2014. p. S155–S155.
• Thakore PI, Ousterout DG, Kabadi AM, Deneke VE, Crawford GE, Reddy TE, et al. CRISPR/Cas9-Based Transcriptional Repressors for Control of Human Gene Expression. In: MOLECULAR THERAPY. NATURE PUBLISHING GROUP; 2014. p. S214–5.
• Schulz E, Bergmann T, Gebbing M, Schildgen V, Schildgen O, Gersbach C, et al. Mutation Detection Following Non-Homologous End Joining (NHEJ): A Comparison of Different Semi Quantitative and Quantitative Methods. In: MOLECULAR THERAPY. NATURE PUBLISHING GROUP; 2014. p. S126–7.
• Thakore PI, Diekman BO, Willard VP, O’Connor S, Brunger JM, Christoforou N, et al. Modulating the Expression of the Cell Cycle Inhibitor p21 to Enhance Cartilage Tissue Engineering With iPSCs. In: MOLECULAR THERAPY. NATURE PUBLISHING GROUP; 2014. p. S157–8.
• High K, Gregory PD, Gersbach C. CRISPR technology for gene therapy. Nature medicine. 2014 May;20(5):476–7.
• Polstein LR, Gersbach CA. Spatiotemporal genetic control of cellular systems. In: Tissue and Organ Regeneration: Advances in Micro- and Nanotechnology. 2014. p. 156–97.
• Gersbach CA, Gaj T, Barbas CF. Comparing genome editing technologies. Genetic Engineering and Biotechnology News. 2014 Mar 1;34(5).
• Brunger JM, Huynh NPT, Guenther CM, Perez-Pinera P, Moutos FT, Sanchez-Adams J, et al. Scaffold-mediated lentiviral transduction for functional tissue engineering of cartilage. Proceedings of the National Academy of Sciences of the United States of America. 2014 Mar;111(9):E798–806.
• Polstein LR, Gersbach CA. Light-inducible gene regulation with engineered zinc finger proteins. Methods in molecular biology (Clifton, NJ). 2014 Jan;1148:89–107.
• Chakraborty S, Ji H, Kabadi AM, Gersbach CA, Christoforou N, Leong KW. CRISPR/Cas9-Based Transcriptional Activation of Endogenous Myod1 to Reprogram Murine Fibroblasts into Skeletal Myocytes. In: MOLECULAR THERAPY. 2014. p. S215–S215.
• Ousterout DG, Perez-Pinera P, Thakore PI, Kabadi AM, Brown MT, Qin X, et al. Erratum: Reading Frame correction by targeted genome editing restores dystrophin expression in cells from duchenne muscular dystrophy patients (Molecular Therapy (2013) 21 (1718-1726) DOI: 10.1038/mt.2013.111). Molecular Therapy. 2013 Nov 1;21(11):2130.
• Perez-Pinera P, Kocak DD, Vockley CM, Adler AF, Kabadi AM, Polstein LR, et al. RNA-guided gene activation by CRISPR-Cas9-based transcription factors. Nat Methods. 2013 Oct;10(10):973–6.
• Ousterout DG, Perez-Pinera P, Thakore PI, Kabadi AM, Brown MT, Qin X, et al. Reading frame correction by targeted genome editing restores dystrophin expression in cells from Duchenne muscular dystrophy patients. Molecular therapy : the journal of the American Society of Gene Therapy. 2013 Sep;21(9):1718–26.
• Gersbach CA. Engineered Proteins for Controlling Gene Expression. 2013 Aug 27;1:125–38.
• Gaj T, Gersbach CA, Barbas CF. ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering. Trends in biotechnology. 2013 Jul;31(7):397–405.
• Perez-Pinera P, Ousterout DG, Brunger JM, Farin AM, Glass KA, Guilak F, et al. Synergistic and Tunable Gene Activation by Combinations of Synthetic Transcription Factors. In: MOLECULAR THERAPY. NATURE PUBLISHING GROUP; 2013. p. S93–S93.
• Dumbauld DW, Lee TT, Singh A, Scrimgeour J, Gersbach CA, Zamir EA, et al. How vinculin regulates force transmission. Proceedings of the National Academy of Sciences of the United States of America. 2013 Jun;110(24):9788–93.
• Glass KA, Brunger JM, Gersbach CA, Guilak F. Tunable expression of IL-1Ra in genetically modified mesenchymal stem cells for cartilage tissue engineering. In: Osteoarthritis and Cartilage. Elsevier BV; 2013. p. S282–3.
• Gibson TM, Gersbach CA. The role of single-cell analyses in understanding cell lineage commitment. Biotechnology journal. 2013 Apr;8(4):397–407.
• Bhakta MS, Henry IM, Ousterout DG, Das KT, Lockwood SH, Meckler JF, et al. Highly active zinc-finger nucleases by extended modular assembly. Genome research. 2013 Mar;23(3):530–8.
• Perez-Pinera P, Ousterout DG, Brunger JM, Farin AM, Glass KA, Guilak F, et al. Synergistic and tunable human gene activation by combinations of synthetic transcription factors. Nat Methods. 2013 Mar;10(3):239–42.
• Tremblay JP, Xiao X, Aartsma-Rus A, Barbas C, Blau HM, Bogdanove AJ, et al. Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases. Molecular therapy : the journal of the American Society of Gene Therapy. 2013 Feb;21(2):266–8.
• Perez-Pinera P, Kocak DD, Vockley CM, Adler AF, Kabadi AM, Polstein LR, et al. RNA-guided gene activation by CRISPR-Cas9-based transcription factors. Nature Methods. 2013;10(10):973–6.
• Duvall CL, Prokop A, Gersbach CA, Davidson JM. Gene Delivery into Cells and Tissues. 2013 Jan 1;687–723.
• Ousterout DG, Perez-Pinera P, Thakore PI, Kabadi AM, Brown MT, Qin X, et al. Reading frame correction by targeted genome editing restores dystrophin expression in cells from duchenne muscular dystrophy patients. Molecular Therapy. 2013;21(9):1718–26.
• Gersbach CA, Barbas CF. Targeted plasmid integration into the Human Genome by engineered recombinases. Topics in Current Genetics. 2013 Jan 1;23:267–84.
• Polstein LR, Gersbach CA. Photoregulated gene expression in human cells with light-inducible engineered transcription factors. ASME 2012 Summer Bioengineering Conference, SBC 2012. 2012 Dec 1;351–2.
• Polstein LR, Gersbach CA. Light-inducible spatiotemporal control of gene activation by customizable zinc finger transcription factors. Journal of the American Chemical Society. 2012 Oct;134(40):16480–3.
• Perez-Pinera P, Ousterout DG, Gersbach CA. Advances in targeted genome editing. Current opinion in chemical biology. 2012 Aug;16(3–4):268–77.
• Brunger JM, Moutos FT, Perez-Pinera P, Lennon DP, Caplan AI, Guilak F, et al. Biomaterial-Mediated Gene Delivery for Tissue Engineering of Articular Cartilage. In: MOLECULAR THERAPY. NATURE PUBLISHING GROUP; 2012. p. S170–S170.
• Perez Pinera P, Ousterout DG, Brown MT, Gersbach CA. Gene targeting to the ROSA26 locus directed by engineered zinc finger nucleases. Nucleic Acids Research. 2012;40(8):3741–52.
• Gersbach CA. Engineered Proteins for Controlling Gene Expression. In: Handbook of Stem Cells, Second Edition: Volume 1-2. 2012. p. 125–38.
• Gersbach CA. Engineered Proteins for Controlling Gene Expression. 2011 Dec 1;159–76.
• Gersbach CA. Engineered bioactive molecules. 2011 Oct 1;5:131–45.
• Gersbach CA, Gaj T, Gordley RM, Mercer AC, Barbas CF. Targeted plasmid integration into the human genome by an engineered zinc-finger recombinase. Nucleic acids research. 2011 Sep;39(17):7868–78.
• Gaj T, Mercer AC, Gersbach CA, Gordley RM, Barbas CF. Structure-guided reprogramming of serine recombinase DNA sequence specificity. Proceedings of the National Academy of Sciences of the United States of America. 2011 Jan 11;108(2):510–5.
• Gaj T, Mercer AC, Gersbach CA, Gordley RM, Barbas CF. Structure-guided reprogramming of serine recombinase DNA sequence specificity. Proceedings of the National Academy of Sciences of the United States of America. 2011 Jan;108(2):498–503.
• Gersbach CA, Gaj T, Gordley RM, Barbas CF. Directed evolution of recombinase specificity by split gene reassembly. Nucleic acids research. 2010 Jul;38(12):4198–206.
• Gersbach CA. Engineered Proteins for Controlling Gene Expression. In: Principles of Regenerative Medicine, Second Edition. 2010. p. 159–76.
• Gordley RM, Gersbach CA, Barbas CF. Synthesis of programmable integrases. Proceedings of the National Academy of Sciences of the United States of America. 2009 Mar;106(13):5053–8.
• Pregizer S, Barski A, Gersbach CA, García AJ, Frenkel B. Identification of novel Runx2 targets in osteoblasts: cell type-specific BMP-dependent regulation of Tram2. Journal of cellular biochemistry. 2007 Dec;102(6):1458–71.
• Gersbach CA, Coyer SR, Le Doux JM, García AJ. Biomaterial-mediated retroviral gene transfer using self-assembled monolayers. Biomaterials. 2007 Dec;28(34):5121–7.
• Gersbach CA, Guldberg RE, García AJ. In vitro and in vivo osteoblastic differentiation of BMP-2- and Runx2-engineered skeletal myoblasts. Journal of cellular biochemistry. 2007 Apr;100(5):1324–36.
• Gersbach CA, Phillips JE, García AJ. Genetic engineering for skeletal regenerative medicine. Annual review of biomedical engineering. 2007 Jan;9:87–119.
• Phillips JE, Gersbach CA, García AJ. Virus-based gene therapy strategies for bone regeneration. Biomaterials. 2007 Jan;28(2):211–29.
• Gersbach CA, Le Doux JM, Guldberg RE, García AJ. Inducible regulation of Runx2-stimulated osteogenesis. Gene therapy. 2006 Jun;13(11):873–82.
• Phillips JE, Gersbach CA, Wojtowicz AM, García AJ. Glucocorticoid-induced osteogenesis is negatively regulated by Runx2/Cbfa1 serine phosphorylation. Journal of cell science. 2006 Feb;119(Pt 3):581–91.
• Gersbach CA, Phillips JE, Guldberg RE, García AJ. Runx2-genetically engineered cells for bone tissue engineering. Proceedings of the 2005 Summer Bioengineering Conference. 2005 Dec 1;2005:603–4.
• Lan MA, Gersbach CA, Michael KE, Keselowsky BG, García AJ. Myoblast proliferation and differentiation on fibronectin-coated self assembled monolayers presenting different surface chemistries. Biomaterials. 2005 Aug;26(22):4523–31.
• Drevs J, Zirrgiebel U, Schmidt-Gersbach CIM, Mross K, Medinger M, Lee L, et al. Soluble markers for the assessment of biological activity with PTK787/ZK 222584 (PTK/ZK), a vascular endothelial growth factor receptor (VEGFR) tyrosine kinase inhibitor in patients with advanced colorectal cancer from two phase I trials. Annals of oncology : official journal of the European Society for Medical Oncology. 2005 Apr;16(4):558–65.
• Gersbach CA, Byers BA, Guldberg RE, Pavlath GK, Garcia AJ. Runx2-stimulated transdifferentiation of primary skeletal myoblasts into an osteoblastic mineralizing phenotype for bone tissue engineering. Transactions - 7th World Biomaterials Congress. 2004 Dec 1;328.
• Gersbach CA, Byers BA, Pavlath GK, García AJ. Runx2/Cbfa1 stimulates transdifferentiation of primary skeletal myoblasts into a mineralizing osteoblastic phenotype. Experimental cell research. 2004 Nov;300(2):406–17.
• Gersbach CA, Byers BA, Pavlath GK, Guldberg RE, García AJ. Runx2/Cbfa1-genetically engineered skeletal myoblasts mineralize collagen scaffolds in vitro. Biotechnology and bioengineering. 2004 Nov;88(3):369–78.
• GERSBACH CA. Runx2/Cbfa1 stimulates transdifferentiation of primary skeletal myoblasts into a mineralizing osteoblastic phenotype. Exp Cell Res. 2004;300:406–17.
• García AJ, Guldberg RE, Byers BA, Gersbach CA, Phillips JE. Addressing cell-sourcing limitations with gene therapy. IEEE engineering in medicine and biology magazine : the quarterly magazine of the Engineering in Medicine & Biology Society. 2003 Sep;22(5):65–70.

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