640 results on '"Cathomen, Toni"'
Search Results
152. Targeted gene addition strategies for the treatment of X-linked lymphoproliferative disease
153. Restoration of progranulin expression rescues cortical neuron generation in an induced pluripotent stem cell model of frontotemporal dementia
154. Gene Editing with Crispr-Cas9 for Treating Beta-Hemoglobinopathies
155. Efficient Recombinase-Mediated Cassette Exchange in hPSCs to Study the Hepatocyte Lineage Reveals AAVS1 Locus-Mediated Transgene Inhibition
156. A novel zinc-finger nuclease platform with a sequence-specific cleavage module
157. Rescue of DNA-PK Signaling and T-Cell Differentiation by Targeted Genome Editing in a prkdc Deficient iPSC Disease Model
158. 483. Epigenetic Silencing of Hepatitis B cccDNA In Vitro and In Vivo Using AAV-Delivered Engineered Repressor Transcription Activator-Like Effector
159. Genome Editing Technologies: Defining a Path to Clinic
160. 118. Inhibition of Hepatitis B Virus Replication In Vivo Following Delivery of Antiviral TALENs With Recombinant Adeno-Associated Viral Vectors
161. Deciphering the impact of parameters influencing transgene expression kinetics after repeated cell transduction with integration-deficient retroviral vectors
162. Restoration of Progranulin Expression Rescues Cortical Neuron Generation in an Induced Pluripotent Stem Cell Model of Frontotemporal Dementia
163. Targeted Integration by Adeno-Associated Virus
164. Autosomal dominant immune dysregulation syndrome in humans with CTLA4 mutations
165. Construction and characterization of adenoviral vectors for the delivery of TALENs into human cells
166. Adenoviral vector DNA for accurate genome editing with engineered nucleases
167. Non-integrating gamma-retroviral vectors as a versatile tool for transient zinc-finger nuclease delivery
168. Efficient Designer Nuclease-Based Homologous Recombination Enables Direct PCR Screening for Footprintless Targeted Human Pluripotent Stem Cells
169. Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases
170. Histone Deacetylase Inhibition Rescues Gene Knockout Levels Achieved with Integrase-Defective Lentiviral Vectors Encoding Zinc-Finger Nucleases
171. Inactivation of Hepatitis B Virus Replication in Cultured Cells and In Vivo with Engineered Transcription Activator-Like Effector Nucleases
172. RNA guides genome engineering
173. Translating the Genomics Revolution: The Need for an International Gene Therapy Consortium for Monogenic Diseases
174. The Nontoxic Cell Cycle Modulator Indirubin Augments Transduction of Adeno-Associated Viral Vectors and Zinc-Finger Nuclease-Mediated Gene Targeting
175. Differential integrity of TALE nuclease genes following adenoviral and lentiviral vector gene transfer into human cells
176. TALE nucleases: tailored genome engineering made easy
177. Retargeting Sleeping Beauty Transposon Insertions by Engineered Zinc Finger DNA-binding Domains
178. Versatile and Efficient Genome Editing in Human Cells by Combining Zinc-Finger Nucleases With Adeno-Associated Viral Vectors
179. Selection-Independent Generation of Gene Knockout Mouse Embryonic Stem Cells Using Zinc-Finger Nucleases
180. A novel zinc-finger nuclease platform with a sequence-specific cleavage module
181. On target? Tracing zinc-finger-nuclease specificity
182. Highly Efficient Zinc-Finger Nuclease-Mediated Disruption of an eGFP Transgene in Keratinocyte Stem Cells without Impairment of Stem Cell Properties
183. Zinc-Finger Nucleases for Somatic Gene Therapy: The Next Frontier
184. Targeted genome editing in pluripotent stem cells using zinc-finger nucleases
185. Zinc-Finger Nuclease Based Genome Surgery: Its All About Specificity
186. Efficient Gene Targeting Mediated by Adeno-Associated Virus and DNA Double-Strand Breaks
187. Matricellular Signaling Molecule CCN1 Attenuates Experimental Autoimmune Myocarditis by Acting as a Novel Immune Cell Migration Modulator
188. Integration Preferences of Wildtype AAV-2 for Consensus Rep-Binding Sites at Numerous Loci in the Human Genome
189. Generation and genetic modification of induced pluripotent stem cells
190. Fate of Recombinant Adeno-Associated Viral Vector Genomes During DNA Double-Strand Break-Induced Gene Targeting in Human Cells
191. Zinc-finger Nuclease-induced Gene Repair With Oligodeoxynucleotides: Wanted and Unwanted Target Locus Modifications
192. Reply to “Genome editing with modularly assembled zinc-finger nucleases”
193. Impaired Axonal Transport in Motor Neurons Correlates with Clinical Prion Disease
194. Ultramicroscopy Reveals Axonal Transport Impairments in Cortical Motor Neurons at Prion Disease
195. Expanding or Restricting the Target Site Repertoire of Zinc-finger Nucleases: The Inter-domain Linker as a Major Determinant of Target Site Selectivity
196. Zinc-finger Nucleases: The Next Generation Emerges
197. Rapid “Open-Source” Engineering of Customized Zinc-Finger Nucleases for Highly Efficient Gene Modification
198. Erratum: Unexpected failure rates for modular assembly of engineered zinc fingers
199. Unexpected failure rates for modular assembly of engineered zinc fingers
200. DNA-binding Specificity Is a Major Determinant of the Activity and Toxicity of Zinc-finger Nucleases
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