A5025290665- CRISPR and Genetic Engineering
- Pluripotent Stem Cells Research
- CAR-T cell therapy research
- Renal and related cancers
- Viral Infections and Immunology Research
- RNA regulation and disease
- Pancreatic function and diabetes
- Immune Cell Function and Interaction
- Glycosylation and Glycoproteins Research
- Cardiac Ischemia and Reperfusion
- Muscle Physiology and Disorders
- Reproductive Biology and Fertility
- RNA Interference and Gene Delivery
- Chromosomal and Genetic Variations
- Cardiac Fibrosis and Remodeling
- T-cell and B-cell Immunology
- Animal Genetics and Reproduction
- Epigenetics and DNA Methylation
- Receptor Mechanisms and Signaling
- Innovative Microfluidic and Catalytic Techniques Innovation
Takeda (Japan)
2020-2021
Kyoto University
2006-2020
Matsumoto University
2020
Howard Hughes Medical Institute
2014-2018
Harvard University
2014-2018
Boston Children's Hospital
2014-2018
Roswell Park Comprehensive Cancer Center
2016
Joslin Diabetes Center
2016
University of Edinburgh
2013
Abstract Prolonged expression of the CRISPR-Cas9 nuclease and gRNA from viral vectors may cause off-target mutagenesis immunogenicity. Thus, a transient delivery system is needed for therapeutic genome editing applications. Here, we develop an extracellular nanovesicle-based ribonucleoprotein named NanoMEDIC by utilizing two distinct homing mechanisms. Chemical induced dimerization recruits Cas9 protein into nanovesicles, then RNA packaging signal self-cleaving riboswitches tether release...
Abstract Genome editing therapy for Duchenne muscular dystrophy (DMD) holds great promise, however, one major obstacle is delivery of the CRISPR-Cas9/sgRNA system to skeletal muscle tissues. In general, AAV vectors are used in vivo delivery, but injections cannot be repeated because neutralization antibodies. Here we report a chemically defined lipid nanoparticle (LNP) which able deliver Cas9 mRNA and sgRNA into by intramuscular injections. Although expressions protein were transient, our...
Abstract Background We previously identified a set of genes called ECATs (ES cell-associated transcripts) that are expressed at high levels in mouse ES cells. Here, we examine the expression and DNA methylation somatic cells germ Results In all examined, promoter region had low cells, but higher contrast, spite their lack pluripotency, male germline stem (GS) most exhibited hypomethylation ECAT regions. observed similar loci adult testis isolated sperm. Some were even less methylated than...
Immune rejection of allogeneic cell therapeutics remains a major problem for immuno-oncology and regenerative medicine. Allogeneic products so far have inferior persistence efficacy when compared with autologous alternatives. Engineering hypoimmune cells may greatly improve their therapeutic benefit. We present new class agonistic immune checkpoint engagers that protect human leukocyte antigen (HLA)-depleted induced pluripotent stem cell-derived endothelial (iECs) from innate cells. Engagers...
The principal factors that lead to proliferation and pluripotency in embryonic stem cells (ESCs) have been vigorously investigated. However, the global network of their full signaling cascade is still unclear. In this study, we found ECAT11 (L1td1) one ESC-associated transcripts harboring a truncated fragment ORF-1, component theL1 retrotransposable element. We generated an knock-in mouse by replacing its coding region with green fluorescent protein. early stage development, fluorescence was...
Abstract The hexose transporter, GLUT2 (SLC2A2), which is expressed by mouse embryos, important for survival before embryonic day 10.5, but its function in embryos unknown. can transport the amino sugar glucosamine (GlcN), could increase substrate hexosamine biosynthetic pathway (HBSP) that produces UDP-N-acetylglucosamine O -linked N-acetylglucosamine modification ( -GlcNAcylation) of proteins. To understand this, we employed a novel murine stem cell (ESC) line that, like expresses...
Adoptive transfer of ex vivo expanded autologous tumor-infiltrating lymphocytes (TILs) can mediate durable and complete responses in significant subsets patients with metastatic melanoma. Major obstacles this approach are the reduced viability transferred T cells, caused by telomere shortening, limited number TILs obtained from patients. Less-differentiated cells long telomeres would be an ideal cell subset for adoptive therapy;however, generating large numbers these less-differentiated is...
Abstract Lymphocytes infiltrating to the injured skeletal muscle have been shown play an important role in regeneration. Site-specific infiltrating/expanding lymphocytes and its molecular features are potential resources for bioengineering such as a drug delivery system. Although some previous studies showed accumulation of muscle, actual frequency distinct lymphocyte clones situ is still unclear. Hence, comprehensive picture repertoire has yet be described detail. To this end, we conducted...