Charles A Gersbach


Affiliation: Duke University Medical Center
Country: USA


  1. Kocak D, Josephs E, Bhandarkar V, Adkar S, Kwon J, Gersbach C. Increasing the specificity of CRISPR systems with engineered RNA secondary structures. Nat Biotechnol. 2019;: pubmed publisher
    ..Our results demonstrate that RNA secondary structure is a fundamental parameter that can tune the activity of diverse CRISPR systems. ..
  2. Nelson C, Wu Y, Gemberling M, Oliver M, Waller M, Bohning J, et al. Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy. Nat Med. 2019;25:427-432 pubmed publisher
    ..This study shows the potential of AAV-CRISPR for permanent genome corrections and highlights aspects of host response and alternative genome editing outcomes that require further study. ..
  3. Glass K, Link J, Brunger J, Moutos F, Gersbach C, Guilak F. Tissue-engineered cartilage with inducible and tunable immunomodulatory properties. Biomaterials. 2014;35:5921-31 pubmed publisher
    ..The ability of functional engineered cartilage to deliver tunable anti-inflammatory cytokines to the joint may enhance the long-term success of therapies for cartilage injuries or osteoarthritis. ..
  4. Kabadi A, Ousterout D, Hilton I, Gersbach C. Multiplex CRISPR/Cas9-based genome engineering from a single lentiviral vector. Nucleic Acids Res. 2014;42:e147 pubmed publisher
    ..This delivery system will be significant to enabling the potential of CRISPR/Cas9-based multiplex genome engineering in diverse cell types. ..
  5. Gibson T, Gersbach C. Single-molecule analysis of myocyte differentiation reveals bimodal lineage commitment. Integr Biol (Camb). 2015;7:663-71 pubmed publisher
    ..These results provide quantitative single-molecule data to support the model of switch-like cell decision making and lineage specification. ..
  6. Gersbach C, Barrangou R. Pulling the genome in opposite directions to dissect gene networks. Genome Biol. 2018;19:42 pubmed publisher
    ..Orthogonal CRISPR-Cas systems have been integrated into combinatorial screens to decipher complex genetic relationships in two recent studies. ..
  7. Kabadi A, Gersbach C. Engineering synthetic TALE and CRISPR/Cas9 transcription factors for regulating gene expression. Methods. 2014;69:188-97 pubmed publisher
    ..We also discuss characteristics of each platform that are best suited for different applications. ..
  8. Black J, Gersbach C. Synthetic transcription factors for cell fate reprogramming. Curr Opin Genet Dev. 2018;52:13-21 pubmed publisher
    ..The rapid development of next-generation technologies with more robust and versatile functionality will continue to expand the application of synthetic transcription factors for cell reprogramming. ..
  9. Robinson Hamm J, Gersbach C. Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy. Hum Genet. 2016;135:1029-40 pubmed publisher
    ..All these approaches to restoring dystrophin expression are encouraging, but many hurdles remain. This review summarizes the current state of these technologies and summarizes considerations for their future development. ..

More Information


  1. Black J, Adler A, Wang H, D Ippolito A, Hutchinson H, Reddy T, et al. Targeted Epigenetic Remodeling of Endogenous Loci by CRISPR/Cas9-Based Transcriptional Activators Directly Converts Fibroblasts to Neuronal Cells. Cell Stem Cell. 2016;19:406-14 pubmed publisher
  2. Klann T, Black J, Gersbach C. CRISPR-based methods for high-throughput annotation of regulatory DNA. Curr Opin Biotechnol. 2018;52:32-41 pubmed publisher
  3. Klann T, Crawford G, Reddy T, Gersbach C. Screening Regulatory Element Function with CRISPR/Cas9-based Epigenome Editing. Methods Mol Biol. 2018;1767:447-480 pubmed publisher
    ..This protocol will be generally useful for implementing genome engineering technologies for high-throughput functional annotation of putative regulatory elements in their native chromosomal context. ..
  4. Nelson C, Hakim C, Ousterout D, Thakore P, Moreb E, Castellanos Rivera R, et al. In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy. Science. 2016;351:403-7 pubmed publisher
    ..This work establishes CRISPR-Cas9-based genome editing as a potential therapy to treat DMD. ..
  5. Thakore P, Kwon J, Nelson C, Rouse D, Gemberling M, Oliver M, et al. RNA-guided transcriptional silencing in vivo with S. aureus CRISPR-Cas9 repressors. Nat Commun. 2018;9:1674 pubmed publisher
    ..In vivo programmable gene silencing enables studies that link gene regulation to complex phenotypes and expands the CRISPR-Cas9 perturbation toolbox for basic research and gene therapy applications. ..
  6. Thakore P, Gersbach C. Design, Assembly, and Characterization of TALE-Based Transcriptional Activators and Repressors. Methods Mol Biol. 2016;1338:71-88 pubmed publisher
    ..These methods for engineering TALE-TFs are useful for studies in reverse genetics and genomics, synthetic biology, and gene therapy. ..
  7. Ousterout D, Gersbach C. The Development of TALE Nucleases for Biotechnology. Methods Mol Biol. 2016;1338:27-42 pubmed publisher
    ..The current status of genome editing and future directions for other uses of these technologies are also discussed. ..
  8. Adkar S, Brunger J, Willard V, Wu C, Gersbach C, Guilak F. Genome Engineering for Personalized Arthritis Therapeutics. Trends Mol Med. 2017;23:917-931 pubmed publisher
    ..We also review the potential contributions of genome engineering in the development of new arthritis therapeutics. ..
  9. Doudna J, Gersbach C. Genome editing: the end of the beginning. Genome Biol. 2015;16:292 pubmed publisher