| 2025 | Long-term correction of hemophilia A via integration of a functionally enhanced FVIII gene into the AAVS1 locus by nickase in patient-derived iPSCs
| EXPERIMENTAL AND MOLECULAR MEDICINE |
| 2024 | Preclinical and dose-ranging assessment of hESC-derived dopaminergic progenitors for a clinical trial on Parkinson's disease
| CELL STEM CELL |
| 2022 | Transplantation of PSA-NCAM-Positive Neural Precursors from Human Embryonic Stem Cells Promotes Functional Recovery in an Animal Model of Spinal Cord Injury | TISSUE ENGINEERING AND REGENERATIVE MEDICINE |
| 2022 | Therapeutic correction of hemophilia A using 2D endothelial cells and multicellular 3D organoids derived from CRISPR/Cas9-engineered patient iPSCs | BIOMATERIALS |
| 2020 | Generation of a human induced pluripotent stem cell line, YCMi002-A, from a Factor VII deficiency patient carrying F7 mutations
| STEM CELL RESEARCH |
| 2019 | Universal Correction of Blood Coagulation Factor VIII in Patient-Derived Induced Pluripotent Stem Cells Using CRISPR/Cas9
| Stem Cell Reports |
| 2019 | Restoration of FVIII expression by targeted gene insertion in the FVIII locus in hemophilia A patient-derived iPSCs
| EXPERIMENTAL AND MOLECULAR MEDICINE |
| 2018 | Wnt signal activation induces midbrain specification through direct binding of the beta-catenin/TCF4 complex to the EN1 promoter in human pluripotent stem cells
| EXPERIMENTAL AND MOLECULAR MEDICINE |
| 2016 | Modeling and correction of structural variations in patient-derived iPSCs using CRISPR/Cas9 | NATURE PROTOCOLS |
| 2016 | Genome-editing technologies for gene correction of hemophilia
| HUMAN GENETICS |
| 2016 | Genome Editing of Structural Variations: Modeling and Gene Correction | TRENDS IN BIOTECHNOLOGY |
| 2015 | Reversion of FMR1 Methylation and Silencing by Editing the Triplet Repeats in Fragile X iPSC-Derived Neurons
| CELL REPORTS |
| 2015 | Functional Correction of Large Factor VIII Gene Chromosomal Inversions in Hemophilia A Patient-Derived iPSCs Using CRISPR-Cas9 | CELL STEM CELL |
| 2015 | PSA-NCAM-negative neural crest cells emerging during neural induction of pluripotent stem cells cause mesodermal tumors and unwanted grafts | STEM CELL REPORTS |
| 2014 | Targeted inversion and reversion of the blood coagulation factor 8 gene in human iPS cells using TALENs
| PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA |
| 2013 | ABCD2 Is a Direct Target of β-Catenin and TCF-4: Implications for X-Linked Adrenoleukodystrophy Therapy
| PLOS ONE |
http://orcid.org/0000-0002-4467-9268
