608 results on '"Porteus, Matthew H"'
Search Results
102. TRACE-Seq Reveals Clonal Reconstitution Dynamics of Gene Targeted Human Hematopoietic Stem Cells
103. High-Efficiency, Selection-free Gene Repair in Airway Stem Cells from Cystic Fibrosis Patients Rescues CFTR Function in Differentiated Epithelia
104. Monocyte lineage-specific glucocerebrosidase expression in human hematopoietic stem cells: A universal genome editing strategy for Gaucher disease
105. Reply to “Efficient Nuclease-free HR by Clade F AAV Requires High MOIs with High Quality Vectors”
106. PLANT BIOTECHNOLOGY: Zinc fingers on target: The existing methods of creating genetically modified plants are inefficient and imprecise. Zinc-finger technology offers the prospect of opening up a swifter and more exact route for crop improvement.
107. Erratum:Correction: Multiplexed genetic engineering of human hematopoietic stem and progenitor cells using CRISPR/Cas9 and AAV6 (eLife (2017) 6 PII: e43690)
108. Highly efficient endogenous human gene correction using designed zinc-finger nucleases
109. Zinc finger nucleases: custom-designed molecular scissors for genome engineering of plant and mammalian cells
110. Chimeric Nucleases Stimulate Gene Targeting in Human Cells
111. Characteristics and Outcome of Children With Beckwith-Wiedemann Syndrome and Wilms’ Tumor: A Report From the National Wilms Tumor Study Group
112. AAV6 Is Superior to Clade F AAVs in Stimulating Homologous Recombination-Based Genome Editing in Human HSPCs
113. Highly efficient editing of the β-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease
114. A New Class of Medicines through DNA Editing
115. Highly Efficient Repair of the ΔF508 Mutation in Airway Stem Cells of Cystic Fibrosis Patients with Functional Rescue of the Differentiated Epithelia
116. Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements
117. Correction: Multiplexed genetic engineering of human hematopoietic stem and progenitor cells using CRISPR/Cas9 and AAV6
118. Small-molecule correctors divert CFTR-F508del from ERAD by stabilizing sequential folding states
119. Global Transcriptional Response to CRISPR/Cas9-AAV6-Based Genome Editing in CD34+ Hematopoietic Stem and Progenitor Cells
120. Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I
121. Uridine Depletion and Chemical Modification Increase Cas9 mRNA Activity and Reduce Immunogenicity without HPLC Purification
122. Priming Human Repopulating Hematopoietic Stem and Progenitor Cells for Cas9/sgRNA Gene Targeting
123. Gene Correction for SCID-X1 in Long-Term Hematopoietic Stem Cells
124. Gene Editing on Center Stage
125. Engineering blood stem cells for autologous transplants for lysosomal diseases: Correction of mucopolysaccharidosis type I using genome-edited hematopoietic stem and progenitor cells
126. Identification of Pre-Existing Adaptive Immunity to Cas9 Proteins in Humans
127. Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells
128. Genome Editing Technologies: Defining a Path to Clinic
129. Correction of X-CGD patient HSPCs by targeted CYBBcDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair
130. Neuronal defects in a human cellular model of 22q11.2 deletion syndrome
131. Multiplexed genetic engineering of human hematopoietic stem and progenitor cells using CRISPR/Cas9 and AAV6
132. Author response: Multiplexed genetic engineering of human hematopoietic stem and progenitor cells using CRISPR/Cas9 and AAV6
133. CRISPR-Mediated Integration of Large Gene Cassettes Using AAV Donor Vectors
134. A Comprehensive TALEN-Based Knockout Library for Generating Human-Induced Pluripotent Stem Cell–Based Models for Cardiovascular Diseases
135. Abstract 100
136. Genotype, Phenotype and T Cell Counts at One Year Predict Survival and Long Term Immune Reconstitution after Transplantation in Severe Combined Immune Deficiency (SCID)—The Primary Immune Deficiency Treatment Consortium (PIDTC)
137. Anti-Fungal Prophylaxis Using Intermediate Dose Ambisome is Associated with Delayed Methotrexate Clearance in Pediatric Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
138. CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells
139. Genome Editing Technologies: Defining a Path to Clinic: Genomic Editing: Establishing Preclinical Toxicology Standards, Bethesda, Maryland 10 June 2014
140. Genetically corrected RAG2-SCID human hematopoietic stem cells restore V(D)J-recombinase and rescue lymphoid deficiency
141. 563. Analysis of DNA Repair Pathway Choice Upon Induction of Double-Strand Breaks by Engineered Nuclease
142. 43. CRISPR/Cas9 and rAAV6-Mediated Targeted Integration at the CCR5 Locus in Hematopoietic Stem and Progenitor Cells
143. 39. FACS-Based Enrichment of a Highly Purified HBB-Targeted Hematopoietic Stem and Progenitor Cell Population Using rAAV6 and CRISPR/Cas9
144. Gene Editing with Crispr-Cas9 for Treating Beta-Hemoglobinopathies
145. Towards a new era in medicine: therapeutic genome editing
146. Initial Results from Stanford Children’s Fanconi Anemia Clinical Trial Using JSP191 Antibody Conditioning and TCRαβ+T-Cell/CD19+B-Cell Depleted Grafts
147. Implementation of a Phase 1b/2a Clinical Trial for Fanconi Anemia Patients Using JSP191 Antibody Conditioning and TCRαβ+T-Cell/CD19+B-Cell Depleted Grafts: A Center Experience
148. Identification of preexisting adaptive immunity to Cas9 proteins in humans
149. Zinc fingers on target: the existing methods of creating genetically modified plants are inefficient and imprecise. Zinc-finger technology offers the prospect of opening up a swifter and more exact route for crop improvement
150. Use of Genome Engineering to Create Patient Specific MLL Translocations in Primary Human Hematopoietic Stem and Progenitor Cells
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