Scholars@Duke
Charles Gersbach

Charles Gersbach

John W. Strohbehn Distinguished Professor of Biomedical Engineering
Biomedical Engineering
Charles.gersbach@duke.edu
Box 90281, 136 Hudson Hall, Durham, NC 27708
2123 CIEMAS, Durham, NC 27708

Featured Works

Targeted Epigenetic Remodeling of Endogenous Loci by CRISPR/Cas9-Based Transcriptional Activators Directly Converts Fibroblasts to Neuronal Cells.

Journal article Cell Stem Cell · September 1, 2016 Featured Publication Overexpression of exogenous fate-specifying transcription factors can directly reprogram differentiated somatic cells to target cell types. Here, we show that similar reprogramming can also be achieved through the direct activation of endogenous genes usin ... Full text Link to item Cite

Genome-editing Technologies for Gene and Cell Therapy.

Journal article Molecular therapy : the journal of the American Society of Gene Therapy · March 2016 Featured Publication Gene therapy has historically been defined as the addition of new genes to human cells. However, the recent advent of genome-editing technologies has enabled a new paradigm in which the sequence of the human genome can be precisely manipulated to achieve a ... Full text Cite

Editing the epigenome: technologies for programmable transcription and epigenetic modulation.

Journal article Nature methods · February 2016 Featured Publication Gene regulation is a complex and tightly controlled process that defines cell identity, health and disease, and response to pharmacologic and environmental signals. Recently developed DNA-targeting platforms, including zinc finger proteins, transcription a ... Full text Cite

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy.

Journal article Science · January 22, 2016 Featured Publication Duchenne muscular dystrophy (DMD) is a devastating disease affecting about 1 out of 5000 male births and caused by mutations in the dystrophin gene. Genome editing has the potential to restore expression of a modified dystrophin gene from the native locus ... Full text Link to item Cite

Highly specific epigenome editing by CRISPR-Cas9 repressors for silencing of distal regulatory elements.

Journal article Nat Methods · December 2015 Featured Publication Epigenome editing with the CRISPR (clustered, regularly interspaced, short palindromic repeats)-Cas9 platform is a promising technology for modulating gene expression to direct cell phenotype and to dissect the causal epigenetic mechanisms of gene regulati ... Full text Link to item Cite

Epigenome editing by a CRISPR-Cas9-based acetyltransferase activates genes from promoters and enhancers.

Journal article Nat Biotechnol · May 2015 Featured Publication Technologies that enable targeted manipulation of epigenetic marks could be used to precisely control cell phenotype or interrogate the relationship between the epigenome and transcriptional control. Here we describe a programmable, CRISPR-Cas9-based acety ... Full text Link to item Cite

A light-inducible CRISPR-Cas9 system for control of endogenous gene activation.

Journal article Nature chemical biology · March 2015 Featured Publication Optogenetic systems enable precise spatial and temporal control of cell behavior. We engineered a light-activated CRISPR-Cas9 effector (LACE) system that induces transcription of endogenous genes in the presence of blue light. This was accomplished by fusi ... Full text Cite

Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy.

Journal article Nat Commun · February 18, 2015 Featured Publication The CRISPR/Cas9 genome-editing platform is a promising technology to correct the genetic basis of hereditary diseases. The versatility, efficiency and multiplexing capabilities of the CRISPR/Cas9 system enable a variety of otherwise challenging gene correc ... Full text Link to item Cite

RNA-guided gene activation by CRISPR-Cas9-based transcription factors.

Journal article Nat Methods · October 2013 Featured Publication Technologies for engineering synthetic transcription factors have enabled many advances in medical and scientific research. In contrast to existing methods based on engineering of DNA-binding proteins, we created a Cas9-based transactivator that is targete ... Full text Link to item Cite