26 results on '"Porteus, Matthew H"'
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2. Transient inhibition of 53BP1 increases the frequency of targeted integration in human hematopoietic stem and progenitor cells
3. Ultra-deep sequencing validates safety of CRISPR/Cas9 genome editing in human hematopoietic stem and progenitor cells
4. Author Correction: Investigation of Cas9 antibodies in the human eye
5. Investigation of Cas9 antibodies in the human eye
6. Gene Editing Rescues In vitro T Cell Development of RAG2-Deficient Induced Pluripotent Stem Cells in an Artificial Thymic Organoid System
7. Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair
8. Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells
9. Neuronal defects in a human cellular model of 22q11.2 deletion syndrome
10. Generation of human striatal organoids and cortico-striatal assembloids from human pluripotent stem cells
11. Metabolic engineering generates a transgene-free safety switch for cell therapy
12. The TRACE-Seq method tracks recombination alleles and identifies clonal reconstitution dynamics of gene targeted human hematopoietic stem cells
13. Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice
14. Identification of preexisting adaptive immunity to Cas9 proteins in humans
15. Engineering monocyte/macrophage−specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing
16. Author Correction: Engineering monocyte/macrophage−specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing
17. Functional significance of U2AF1 S34F mutations in lung adenocarcinomas
18. Gene correction for SCID-X1 in long-term hematopoietic stem cells
19. Efficient scarless genome editing in human pluripotent stem cells
20. A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells
21. CRISPR/Cas9 genome editing in human hematopoietic stem cells
22. Correction to: Gene Editing Rescues in Vitro T Cell Development of RAG2-Deficient Induced Pluripotent Stem Cells in an Artificial Thymic Organoid System
23. Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I
24. Author Correction: Gene correction for SCID-X1 in long-term hematopoietic stem cells
25. CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells
26. Genome Editing of the Blood: Opportunities and Challenges
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