Patent classifications
C12N2760/18843
Gene expression system using stealthy RNA, and gene introduction/expression vector including said RNA
The present invention enables simultaneous and stable expression of a plurality of foreign genes by using a stealthy RNA gene expression system that is a complex that does not activate the innate immune mechanism and is formed from an RNA-dependent RNA polymerase, a single-strand RNA binding protein, and negative-sense single-strand RNAs including the following (1) to (8): (1) a target RNA sequence that codes for any protein or functional RNA; (2) an RNA sequence forming a noncoding region and derived from mRNA expressed in animal cells; (3) a transcription initiation signal sequence recognized by the RNA-dependent RNA polymerase; (4) a transcription termination signal sequence recognized by the polymerase; (5) an RNA sequence containing a replication origin recognized by the polymerase; (6) an RNA sequence that codes for the polymerase and of which codons are optimized for the species from which an introduction target cell is derived; (7) an RNA sequence that codes for a protein for regulating the activity of the polymerase and of which codons are optimized for the species from which the introduction target cell is derived; and (8) an RNA sequence that codes for the single-strand RNA binding protein and of which codons are optimized for the species from which the introduction target cell is derived.
Method for Producing Naive Human IPS Cells From Somatic Cells
Provided is a method for producing naive induced pluripotent stem cells from human somatic cells, comprising the following steps (1) to (3): (1) introducing one or more vectors containing a reprogramming factor into human somatic cells, (2) culturing said somatic cells in the presence of a naive medium, and (3) after step (2), culturing the resulting cells in the presence of the naive medium under the condition in which the amount of the vectors per the somatic cell is reduced to 30% or less of that at the start of step 3.
GENERATION OF INDUCED PLURIPOTENT STEM CELL LINES FROM HUMAN PATIENTS WITH MUTATIONS IN THE GLUCOKINASE GENE
Heterozygous and homozygous mutations in the glucokinase (GCK) gene lead to maturity-onset diabetes of the young type 2 (MODY2) and permanent neonatal diabetes (PNDM), respectively. The present invention relates to a method for generating induced pluripotent stem cell (iPSC) lines from patients with MODY2 and PNDM due to mutations in the GCK gene. The generated iPSC lines are able to differentiate into the three germ layers and show normal karyotypes. These iPSC lines can serve as valuable human cell models for understanding diabetes pathogenesis and developing new therapies for diabetes.
USES OF INDUCED NEURAL STEM CELLS DERIVED FROM PERIPHERAL BLOOD MONONUCLEAR CELLS
A method of treating neurodegenerative diseases or disorders, especially Parkinson's disease and a method of inducing neural stem cells from peripheral blood mononuclear cells. The induced neural stem cells can express neural stem cell-related genes and differentiate into neurons, astrocytes and oligodendrocytes. The dopaminergic precursors derived from the induced neural stem cells are transplanted into the striatum of the PD mouse models without any sign of tumorigenesis, thereby improving the behaviors of the PD mouse models and slowing down the progression of Parkinson's disease.
Materials and methods for the manufacture of pluripotent stem cells
Provided here in are methods of producing induced pluripotent stem cells (iPSCs) and isolated population of produced induced pluripotent stem cells (iPSCs). Also provided herein are methods of treating a subject in need thereof using the produced iPSCs or pharmaceutical compositions comprising the produced iPSCs.
PLATFORM FOR THE INDUCTION & MAINTENANCE OF GROUND STATE PLURIPOTENCY
The invention provides compositions and methods for manufacturing pluripotent cells. In particular, the invention provides improved culture platforms for manufacturing pluripotent cells with ground state pluripotency.
OPTIMIZED HIV ENVELOPE GENE AND EXPRESSION THEREOF
The present invention relates to a vector(s) containing and expressing an optimized HIV EnvF gene, methods for making the same and cell substrates qualified for vaccine production which may comprise vector(s) containing optimized HIV genes.
Modified Sendai virus vaccine and imaging vector
The present invention relates to a Sendai virus or recombinant Sendai virus vector. In particular the present invention provides methods, vectors, formulations, compositions, and kits for a modified Enders strain Sendai viral vector. An immunogenic vector can be used in any in vitro or in vivo system. Moreover, some embodiments include vectors for imaging virus growth, location and transmission.
Optimized HIV envelope gene and expression thereof
The present invention relates to a vector(s) containing and expressing an optimized HIV EnvF gene, methods for making the same and cell substrates qualified for vaccine production which may comprise vector(s) containing optimized HIV genes.
HIGHLY FUNCTIONAL HEPATOCYTE AND USE THEREOF
The object is to obtain a highly functional hepatocyte that has a high ammonia-metabolizing function and is theoretically able to infinitely proliferate from a human iPS cell. There is provided a method for preparing an artificial hepatocyte comprising the following steps: the differentiation induction step 1 of culturing an iPS cell in a differentiation induction medium I containing activin A; the differentiation induction step 2 of culturing a cell obtained in the differentiation induction step 1 in a differentiation induction medium II containing bone morphogenetic protein 4 (BMP4) and fibroblast growth factor 2 (FGF2); and the differentiation induction step 3 of culturing a cell obtained in the differentiation induction step 2 in a differentiation induction medium III containing hepatocyte growth factor (HGF), oncostatin M, dexamethasone, and N,N-(methylenebis)(4,1-phenylene)diacetamide (FH1) and/or 2-([N-(5-chloro-2-methylphenyl)(methylsulfonamido)-N-(2,6-difluorophenyl)acetamide (FPH1) to obtain an artificial hepatocyte. The obtained artificial hepatocyte has an ammonia-metabolizing function of 100 ?g/dl/24 h or higher, and can constitute a mesh structure.