Publications
Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy." Human genetics 135, no. 9 (2016): 1029-1040.
"Genome-editing Technologies for Gene and Cell Therapy." Molecular therapy : the journal of the American Society of Gene Therapy 24, no. 3 (2016): 430-446.
"In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy." Science 351, no. 6271 (2016): 403-407.
"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 24, no. 4 (2016): 697-706.
"N-cadherin is Key to Expression of the Nucleus Pulposus Cell Phenotype under Selective Substrate Culture Conditions." Scientific reports 6 (2016).
"Targeted Epigenetic Remodeling of Endogenous Loci by CRISPR/Cas9-Based Transcriptional Activators Directly Converts Fibroblasts to Neuronal Cells." Cell Stem Cell 19, no. 3 (2016): 406-414.
"59. Multiplex Gene Activation by CRISPR/Cas9-Based Transcription Factors for the Direct Conversion of Fibroblasts to a Neuronal Phenotype." In Molecular Therapy, S26. Vol. 23. 2015.
"Anatomically-Shaped Tissue-Engineered Cartilage with Tunable and Inducible Anti-Inflammatory Capabilities." In TISSUE ENGINEERING PART A, S330. Vol. 21. 2015.
"Correction of dystrophin expression in cells from Duchenne muscular dystrophy patients through genomic excision of exon 51 by zinc finger nucleases." Mol Ther 23, no. 3 (2015): 523-532.
"Correction of Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients Through Genomic Excision of Exon 51 by Zinc Finger Nucleases." Molecular Therapy 23, no. 3 (2015): 523-532.
"CRISPRi Immunomodulation for Tissue Engineering/Stem Cell Therapies Targeting Intervertebral Disc Degeneration." In TISSUE ENGINEERING PART A, S170. Vol. 21. 2015.
"Enabling functional genomics with genome engineering." Genome research 25, no. 10 (2015): 1442-1455.
"Enhanced MyoD-induced transdifferentiation to a myogenic lineage by fusion to a potent transactivation domain." ACS Synth Biol 4, no. 6 (2015): 689-699.
"Epigenome editing by a CRISPR-Cas9-based acetyltransferase activates genes from promoters and enhancers." Nat Biotechnol 33, no. 5 (2015): 510-517.
"Genome editing: the end of the beginning." Genome biology 16 (2015).
"Genome-wide specificity of DNA binding, gene regulation, and chromatin remodeling by TALE- and CRISPR/Cas9-based transcriptional activators." Genome Res 25, no. 8 (2015): 1158-1169.
"Highly specific epigenome editing by CRISPR-Cas9 repressors for silencing of distal regulatory elements." Nat Methods 12, no. 12 (2015): 1143-1149.
"Knockdown of the cell cycle inhibitor p21 enhances cartilage formation by induced pluripotent stem cells." Tissue engineering. Part A 21, no. 7-8 (2015): 1261-1274.
"A light-inducible CRISPR-Cas9 system for control of endogenous gene activation." Nature Chemical Biology 11, no. 3 (2015): 198-200.
"Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy." Nat Commun 6 (2015): 6244-.
"Multiplex Gene Activation by CRISPR/Cas9-Based Transcription Factors for the Direct Conversion of Fibroblasts to a Neuronal Phenotype." In Molecular Therapy, S26. Vol. 23. 2015.
"Regulation of chromatin accessibility and Zic binding at enhancers in the developing cerebellum." Nat Neurosci 18, no. 5 (2015): 647-656.
"Single-molecule analysis of myocyte differentiation reveals bimodal lineage commitment." Integrative biology : quantitative biosciences from nano to macro 7, no. 6 (2015): 663-671.
"Structure and specificity of the RNA-guided endonuclease Cas9 during DNA interrogation, target binding and cleavage." Nucleic acids research 43, no. 18 (2015): 8924-8941.
"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 18, no. 8 (2014): 835-839.
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