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Rapid "open-source" engineering of customized zinc-finger nucleases for highly efficient gene modificationA matrix-less measles virus is infectious and elicits extensive cell fusion: consequences for propagation in the brain.Autosomal dominant immune dysregulation syndrome in humans with CTLA4 mutationsStructure-based redesign of the dimerization interface reduces the toxicity of zinc-finger nucleasesImproved bi-allelic modification of a transcriptionally silent locus in patient-derived iPSC by Cas9 nickaseTALENs facilitate targeted genome editing in human cells with high specificity and low cytotoxicityA functional complex of adenovirus proteins E1B-55kDa and E4orf6 is necessary to modulate the expression level of p53 but not its transcriptional activity.A genetic screen identifies a cellular regulator of adeno-associated virus.Measles viruses with altered envelope protein cytoplasmic tails gain cell fusion competence.Selection-independent generation of gene knockout mouse embryonic stem cells using zinc-finger nucleases.Novel lentiviral vectors with mutated reverse transcriptase for mRNA delivery of TALE nucleasesA novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity.Engineered zinc finger nickases induce homology-directed repair with reduced mutagenic effectsAutonomous zinc-finger nuclease pairs for targeted chromosomal deletion.Editing CCR5: a novel approach to HIV gene therapy.TALE-PvuII fusion proteins--novel tools for gene targeting.Rescue of DNA-PK Signaling and T-Cell Differentiation by Targeted Genome Editing in a prkdc Deficient iPSC Disease Model.Versatile and efficient genome editing in human cells by combining zinc-finger nucleases with adeno-associated viral vectors.Targeted Integration of a Super-Exon into the CFTR Locus Leads to Functional Correction of a Cystic Fibrosis Cell Line Model.Retargeting transposon insertions by the adeno-associated virus Rep protein.Efficient Recombinase-Mediated Cassette Exchange in hPSCs to Study the Hepatocyte Lineage Reveals AAVS1 Locus-Mediated Transgene InhibitionRetargeting sleeping beauty transposon insertions by engineered zinc finger DNA-binding domains.Streptococcus thermophilus CRISPR-Cas9 Systems Enable Specific Editing of the Human GenomeDifferential integrity of TALE nuclease genes following adenoviral and lentiviral vector gene transfer into human cells.Genotoxicity in gene therapy: an account of vector integration and designer nucleases.Inactivation of hepatitis B virus replication in cultured cells and in vivo with engineered transcription activator-like effector nucleases.Ultramicroscopy reveals axonal transport impairments in cortical motor neurons at prion diseaseHistone deacetylase inhibition rescues gene knockout levels achieved with integrase-defective lentiviral vectors encoding zinc-finger nucleases.Zinc-finger nuclease based genome surgery: it's all about specificity.TALE nucleases: tailored genome engineering made easy.Efficient designer nuclease-based homologous recombination enables direct PCR screening for footprintless targeted human pluripotent stem cells.Proven and novel strategies for efficient editing of the human genome.Non-integrating gamma-retroviral vectors as a versatile tool for transient zinc-finger nuclease delivery.Therapeutic genome editing with engineered nucleases.TALEN-mediated functional correction of X-linked chronic granulomatous disease in patient-derived induced pluripotent stem cells.Restoration of progranulin expression rescues cortical neuron generation in an induced pluripotent stem cell model of frontotemporal dementia.Adenoviral vector DNA for accurate genome editing with engineered nucleases.Refining strategies to translate genome editing to the clinic.The nontoxic cell cycle modulator indirubin augments transduction of adeno-associated viral vectors and zinc-finger nuclease-mediated gene targeting.Expanding or restricting the target site repertoire of zinc-finger nucleases: the inter-domain linker as a major determinant of target site selectivity.
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0000-0002-7757-4630