A5019048819- CRISPR and Genetic Engineering
- RNA and protein synthesis mechanisms
- Pluripotent Stem Cells Research
- Mesenchymal stem cell research
- RNA Interference and Gene Delivery
- CAR-T cell therapy research
- Virus-based gene therapy research
- Advanced biosensing and bioanalysis techniques
- RNA regulation and disease
- Angiogenesis and VEGF in Cancer
- Animal Genetics and Reproduction
- Innovation and Socioeconomic Development
- Bone Tissue Engineering Materials
- Ubiquitin and proteasome pathways
- Food Quality and Safety Studies
- Retinal Development and Disorders
- Radiation Effects in Electronics
- Low-power high-performance VLSI design
- Parkinson's Disease Mechanisms and Treatments
- Pancreatic function and diabetes
- Semiconductor materials and devices
- Lysosomal Storage Disorders Research
- Nutrition, Health and Food Behavior
- Tissue Engineering and Regenerative Medicine
- Plant Virus Research Studies
Yonsei University
2016-2025
Institute for Basic Science
2017-2024
Yonsei University Health System
2024
University of California, San Francisco
2023
ToolGen (South Korea)
2014-2023
Severance Hospital
2019-2022
ORCID
2021
Seoul National University
2009-2019
National Medical Center
2017-2018
Korea Research Institute of Chemical Technology
2010-2017
The combined availability of whole genome sequences and editing tools is set to revolutionize the field fruit biotechnology by enabling introduction targeted genetic changes with unprecedented control accuracy, both explore emergent phenotypes introduce new functionalities. Although plasmid-mediated delivery components plant cells very efficient, it also presents some drawbacks, such as possible random integration plasmid in host genome. Additionally, may well be intercepted current...
Abstract Several CRISPR-Cas9 orthologues have been used for genome editing. Here, we present the smallest Cas9 orthologue characterized to date, derived from Campylobacter jejuni (CjCas9), efficient editing in vivo . After determining protospacer-adjacent motif (PAM) sequences and optimizing single-guide RNA (sgRNA) length, package CjCas9 gene, its sgRNA sequence, a marker gene an all-in-one adeno-associated virus (AAV) vector produce resulting at high titer. is highly specific, cleaving...
RNA-guided endonucleases (RGENs) derived from the CRISPR/Cas system represent an efficient tool for genome editing. RGENs consist of two components: Cas9 protein and guide RNA. Plasmid-mediated delivery these components into cells can result in uncontrolled integration plasmid sequence host genome, unwanted immune responses potential safety problems that be caused by bacterial sequences. Furthermore, this method requires transfection tools. Here we show simple treatment with cell-penetrating...
Abstract The use of CRISPR-Cas9 as a therapeutic reagent is hampered by its off-target effects. Although rationally designed S. pyogenes Cas9 (SpCas9) variants that display higher specificities than the wild-type SpCas9 protein are available, these attenuated often poorly efficient in human cells. Here, we develop directed evolution approach E. coli to obtain Sniper-Cas9, which shows high without killing on-target activities Unlike other engineered variants, Sniper-Cas9 WT-level with...
Broad applications of zinc finger nuclease (ZFN) technology—which allows targeted genome editing—in research, medicine, and biotechnology are hampered by the lack a convenient, rapid, publicly available method for synthesis functional ZFNs. Here we describe an efficient easy-to-practice modular-assembly using fingers to make ZFNs that can modify DNA sequences predetermined genomic sites in human cells. We synthesized tested hundreds target dozens different CCR5 gene—a co-receptor required...
Abstract The efficacy of T-cell therapy is inhibited by various tumor-associated immunosuppressive ligands and soluble factors. Such inhibitory signals turn specific signaling pathways on or off, impeding the anticancer functions T cells. Many studies have focused PD-1 CTLA-4 blockade to invigorate through CD28/B7 signaling, but obtaining robust clinical outcomes remains challenging. In this study, we use CRISPR/Cas9 potentiate function increasing CD3 via knockout diacylglycerol kinase...
We evaluated SpCas9 activities at 12,832 target sequences using a high-throughput approach based on human cell library containing single-guide RNA-encoding and sequence pairs. Deep learning-based training this large dataset of SpCas9-induced indel frequencies led to the development activity-predicting model named DeepSpCas9. When tested against independently generated datasets (our own those published by other groups), DeepSpCas9 showed high generalization performance. is available...
Hemophilia is a hereditary disease that remains incurable. Although innovative treatments such as gene therapy or bispecific antibody have been introduced, substantial unmet needs still exist with respect to achieving long-lasting therapeutic effects and treatment options for inhibitor patients. Antithrombin (AT), an endogenous negative regulator of thrombin generation, potent genome editing target sustainable patients hemophilia A B. In this study, we developed optimized lipid nanoparticles...
Signal transduction within the canonical Wnt/beta-catenin pathway drives development and carcinogenesis through programmed or unprogrammed changes in gene transcription. Although upstream events linked to signal-induced activation of beta-catenin cytoplasm have been deciphered considerable detail, much less is known regarding mechanism by which stimulates target transcription nucleus. Here, we show that physically functionally targets MED12 subunit Mediator activate The transactivation...
Despite the recent discoveries of and interest in numerous structural variations (SVs)—which include duplications inversions—in human other higher eukaryotic genomes, little is known about etiology biology these SVs, partly due to lack molecular tools with which create individual SVs cultured cells model organisms. Here, we present a novel method inducing inversions targeted manner without pre-manipulation genome. We found that zinc finger nucleases (ZFNs) designed target two different sites...
Abstract Mitochondrial division is critical for the maintenance and regulation of mitochondrial function, quality distribution. This process controlled by cytosolic actin-based constriction machinery dynamin-related protein 1 (Drp1) on outer membrane (OMM). Although physiology, including oxidative phosphorylation, also important efficient division, morphological alterations inner-membrane (IMM) have not been clearly elucidated. Here we report spontaneous repetitive inner compartment (CoMIC)...
CD49f (integrin subunit α6) regulates signaling pathways in a variety of cellular activities. However, the role regulating differentiation and pluripotency stem cells has not been fully investigated. Therefore, this study, human mesenchymal (hMSCs) were induced to form spheres under nonadherent culture conditions, we found that CD49f‐positive population was enriched MSC compared with MSCs monolayer. The expression regulated ability hMSCs associated an activation phosphatidylinositol 3‐kinase...