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Proximity analysis of native proteomes reveals phenotypic modifiers in a mouse model of autism and related neurodevelopmental conditions.
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- Nature Communications, 2024, v. 15, n. 1, p. 1, doi. 10.1038/s41467-024-51037-x
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Epigenome editing technologies for discovery and medicine.
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- Nature Biotechnology, 2024, v. 42, n. 8, p. 1199, doi. 10.1038/s41587-024-02320-1
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A humanized mouse model for adeno-associated viral gene therapy.
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- Nature Communications, 2024, v. 15, n. 1, p. 1, doi. 10.1038/s41467-024-46017-0
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Cross-species evolution of a highly potent AAV variant for therapeutic gene transfer and genome editing.
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- Nature Communications, 2022, v. 13, n. 1, p. 1, doi. 10.1038/s41467-022-33745-4
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- Article
Respiratory Pathology in a Humanized Mouse Model of Duchenne Muscular Dystrophy.
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- FASEB Journal, 2021, v. 35, p. N.PAG, doi. 10.1096/fasebj.2021.35.S1.03516
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Branched-chain α-ketoacids are preferentially reaminated and activate protein synthesis in the heart.
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- Nature Communications, 2021, v. 12, n. 1, p. 1, doi. 10.1038/s41467-021-21962-2
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The once and future gene therapy.
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- Nature Communications, 2020, v. 11, n. 1, p. N.PAG, doi. 10.1038/s41467-020-19505-2
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Enhancer RNAs predict enhancer–gene regulatory links and are critical for enhancer function in neuronal systems.
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- Nucleic Acids Research, 2020, v. 48, n. 17, p. 9550, doi. 10.1093/nar/gkaa671
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Prospective isolation of chondroprogenitors from human iPSCs based on cell surface markers identified using a CRISPR-Cas9-generated reporter.
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- Stem Cell Research & Therapy, 2020, v. 11, n. 1, p. 1, doi. 10.1186/s13287-020-01597-8
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Targeted transcriptional modulation with type I CRISPR–Cas systems in human cells.
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- Nature Biotechnology, 2019, v. 37, n. 12, p. 1493, doi. 10.1038/s41587-019-0235-7
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Genome-wide CRISPR Screen to Identify Genes that Suppress Transformation in the Presence of Endogenous Kras<sup>G12D</sup>.
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- Scientific Reports, 2019, v. 9, n. 1, p. N.PAG, doi. 10.1038/s41598-019-53572-w
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An anionic, endosome-escaping polymer to potentiate intracellular delivery of cationic peptides, biomacromolecules, and nanoparticles.
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- Nature Communications, 2019, v. 10, n. 1, p. 1, doi. 10.1038/s41467-019-12906-y
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Jumping at the chance for precise DNA integration.
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- Nature Biotechnology, 2019, v. 37, n. 9, p. 1004, doi. 10.1038/s41587-019-0210-3
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Increasing the specificity of CRISPR systems with engineered RNA secondary structures.
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- Nature Biotechnology, 2019, v. 37, n. 6, p. 657, doi. 10.1038/s41587-019-0095-1
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RNA-guided transcriptional silencing in vivo with S. aureus CRISPR-Cas9 repressors.
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- Nature Communications, 2018, v. 9, n. 1, p. 1, doi. 10.1038/s41467-018-04048-4
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Pulling the genome in opposite directions to dissect gene networks.
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- Genome Biology, 2018, v. 19, p. 1, doi. 10.1186/s13059-018-1425-1
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Bidirectional approaches for optogenetic regulation of gene expression in mammalian cells using Arabidopsis cryptochrome 2.
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- Nucleic Acids Research, 2017, v. 45, n. 20, p. 1, doi. 10.1093/nar/gkx260
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Incomplete MyoD-induced transdifferentiation is associated with chromatin remodeling deficiencies.
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- Nucleic Acids Research, 2017, v. 45, n. 20, p. 11684, doi. 10.1093/nar/gkx773
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- Article
Generation and comparison of CRISPR-Cas9 and Cre-mediated genetically engineered mouse models of sarcoma.
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- Nature Communications, 2017, v. 8, n. 7, p. 15999, doi. 10.1038/ncomms15999
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CRISPR-Cas9 epigenome editing enables high-throughput screening for functional regulatory elements in the human genome.
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- Nature Biotechnology, 2017, v. 35, n. 6, p. 561, doi. 10.1038/nbt.3853
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CRISPR/Cas9 Editing of Murine Induced Pluripotent Stem Cells for Engineering Inflammation-Resistant Tissues.
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- Arthritis & Rheumatology, 2017, v. 69, n. 5, p. 1111, doi. 10.1002/art.39982
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Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy.
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- Human Genetics, 2016, v. 135, n. 9, p. 1029, doi. 10.1007/s00439-016-1725-z
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N-cadherin is Key to Expression of the Nucleus Pulposus Cell Phenotype under Selective Substrate Culture Conditions.
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- Scientific Reports, 2016, p. 28038, doi. 10.1038/srep28038
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Cas9 loosens its grip on off-target sites.
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- Nature Biotechnology, 2016, v. 34, n. 3, p. 298, doi. 10.1038/nbt.3501
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Genome editing: the end of the beginning.
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- Genome Biology, 2015, v. 16, p. 1, doi. 10.1186/s13059-015-0860-5
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Structure and specificity of the RNA-guided endonuclease Cas9 during DNA interrogation, target binding and cleavage.
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- Nucleic Acids Research, 2015, v. 43, n. 18, p. 8924, doi. 10.1093/nar/gkv892
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Single-molecule analysis of myocyte differentiation reveals bimodal lineage commitment.
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- Integrative Biology, 2015, v. 7, n. 6, p. 663, doi. 10.1039/c5ib00057b
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Epigenome editing by a CRISPR-Cas9-based acetyltransferase activates genes from promoters and enhancers.
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- Nature Biotechnology, 2015, v. 33, n. 5, p. 510, doi. 10.1038/nbt.3199
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Regulation of chromatin accessibility and Zic binding at enhancers in the developing cerebellum.
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- Nature Neuroscience, 2015, v. 18, n. 5, p. 647, doi. 10.1038/nn.3995
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- Article
Correction of Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients Through Genomic Excision of Exon 51 by Zinc Finger Nucleases.
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- Molecular Therapy, 2015, v. 23, n. 3, p. 523, doi. 10.1038/mt.2014.234
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A light-inducible CRISPR-Cas9 system for control of endogenous gene activation.
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- Nature Chemical Biology, 2015, v. 11, n. 3, p. 198, doi. 10.1038/nchembio.1753
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Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy.
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- Nature Communications, 2015, v. 6, n. 2, p. 6244, doi. 10.1038/ncomms7244
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Vector modifications to eliminate transposase expression following piggyBac-mediated transgenesis.
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- Scientific Reports, 2014, p. 1, doi. 10.1038/srep07403
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Multiplex CRISPR/Cas9-based genome engineering from a single lentiviral vector.
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- Nucleic Acids Research, 2014, v. 42, n. 19, p. e147, doi. 10.1093/nar/gku749
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Genome engineering: the next genomic revolution.
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- Nature Methods, 2014, v. 11, n. 10, p. 1009, doi. 10.1038/nmeth.3113
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RNA-guided gene activation by CRISPR-Cas9-based transcription factors.
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- Nature Methods, 2013, v. 10, n. 10, p. 973, doi. 10.1038/nmeth.2600
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Reading Frame Correction by Targeted Genome Editing Restores Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients.
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- Molecular Therapy, 2013, v. 21, n. 9, p. 1718, doi. 10.1038/mt.2013.111
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The role of single-cell analyses in understanding cell lineage commitment.
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- Biotechnology Journal, 2013, v. 8, n. 4, p. 397, doi. 10.1002/biot.201200201
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Synergistic and tunable human gene activation by combinations of synthetic transcription factors.
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- Nature Methods, 2013, v. 10, n. 3, p. 239, doi. 10.1038/nmeth.2361
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Gene targeting to the ROSA26 locus directed by engineered zinc finger nucleases.
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- Nucleic Acids Research, 2012, v. 40, n. 8, p. 3741, doi. 10.1093/nar/gkr1214
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Targeted plasmid integration into the human genome by an engineered zinc-finger recombinase.
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- Nucleic Acids Research, 2011, v. 39, n. 17, p. 7868, doi. 10.1093/nar/gkr421
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Directed evolution of recombinase specificity by split gene reassembly.
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- Nucleic Acids Research, 2010, v. 38, n. 12, p. 4198, doi. 10.1093/nar/gkq125
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- Article
Identification of novel Runx2 targets in osteoblasts: Cell type-specific BMP-dependent regulation of Tram2.
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- Journal of Cellular Biochemistry, 2007, v. 102, n. 6, p. 1458, doi. 10.1002/jcb.21366
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In vitro and in vivo osteoblastic differentiation of BMP-2- and Runx2-engineered skeletal myoblasts.
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- Journal of Cellular Biochemistry, 2007, v. 100, n. 5, p. 1324, doi. 10.1002/jcb.21118
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577. Inducible Regulation of Runx2-Stimulated Osteogenesis.
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- Molecular Therapy, 2006, v. 13, p. S222, doi. 10.1016/j.ymthe.2006.08.650
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814. Biomaterial-Mediated Retroviral Gene Delivery.
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- Molecular Therapy, 2006, v. 13, p. S315, doi. 10.1016/j.ymthe.2006.08.899
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Runx2/Cbfa1-genetically engineered skeletal myoblasts mineralize collagen scaffolds in vitro.
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- Biotechnology & Bioengineering, 2004, v. 88, n. 3, p. 369, doi. 10.1002/bit.20251
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