Abstract: The disclosure relates to human mesenchymal stem cells from Wharton's jelly, a method for preparing them, and the therapeutic uses thereof, in particular for the treatment of sepsis and specifically septic shock. The treatment may involve the administration to a subject in need thereof a pharmaceutical composition including the human mesenchymal stem cells from Wharton's jelly and a pharmaceutically acceptable excipient.

Abstract: The presently disclosed subject matter provides for in vitro methods of inducing differentiation of human stem cells into neural crest, cranial placode or non-neuro ectoderm precursors, and cells generated by such methods. The presently disclosed subject matter also provides for uses of such cells for treating neurodegenerative and pituitary disorders.

Abstract: A culture medium is provided which is capable of establishing expanded potential stem cell (EPSC) lines which resemble naïve or ground state ES cells, but are also able to differentiate into placenta trophoblasts and the embryo proper. Methods are provided using the medium for the in vitro conversion and maintenance of cells, including pluripotent cells into EPSCs.

Abstract: A method of measuring immunocompetence is described. This method provides a means for assessing the effects of diseases or conditions that compromise the immune system and of therapies aimed to reconstitute it. This method is based on quantifying T-cell diversity by calculating the number of diverse T-cell receptor (TCR) beta chain variable regions from blood cells.

Abstract: Provided is a method of differentiating a pluripotent stem cell of mammalian origin into a desired cell type by predicting the direction of cell differentiation to be caused by induction of expression of a transcription factor. A human gene expression correlation matrix using human cells has been newly created, and further, it has been confirmed that human pluripotent stem cells can be differentiated into a desired cell type by introducing, into the human pluripotent stem cells, a transcription factor cocktail selected from the matrix.

Abstract: The present invention relates to genetically engineered non-human mammal and cells, organs and tissues; to use thereof in medicinal and disease research; to a method for producing non-human animals and cells, organs and tissues; and to a method for researching in medicine and disease by virtue of the non-human mammals or cells, organs or tissues.

Abstract: Heart failure with preserved ejection fraction (HFpEF) is a disease condition characterized by heart failure (HF) signs and symptoms, but with normal or near normal left ventricular ejection fraction (LVEF) and is not responsive to standard therapy for treatment of HF. Described herein are compositions and methods related to use of cardiosphere derived cells (CDCs) and their exosomes to improve left ventricular structure, function and overall outcome. Administration of CDCs led to improved LV relaxation, lower LV end-diastolic pressure, decreased lung congestion and enhanced survival. Lower risk of arrhythmias in HFpEF was also observed following CDC administration. Improvement of diastolic dysfunction following administration of CDC-derived exosomes was observed, along with decreased mortality. In view of these salutary effects, CDCs and CDC-derived exosomes are beneficial in the treatment of HFpEF.

Abstract: The present invention is directed to compositions and methods for treating aromatic L-amino acid decarboxylase (AADC) deficiency. This invention includes a method of treating AADC deficiency in a pediatric subject, comprising the steps of: (a) providing a pharmaceutical formulation comprising an rAAV2-hAADC vector, (b) stereotactically delivering the pharmaceutical formulation to at least one target site in the brain of the subject in a dose of an amount at least about 1.8×1011 vg; wherein delivering the pharmaceutical formulation to the brain is optionally by frameless stereotaxy, and optionally wherein the dose is an amount of at least about 2.4×1011 vg and in some embodiments wherein the pharmaceutical formulation comprises a rAAV2-hAADC vector concentration of about 5.7×1011 vg/mL.

Abstract: An object of the present invention is to prepare a functional intestinal organoid from pluripotent stem cells. An intestinal organoid is prepared from pluripotent stem cells, by the following steps (1) to (4): (1) differentiating pluripotent stem cells into endoderm-like cells; (2) differentiating the endoderm-like cells obtained in step (1) into intestinal stem cell-like cells; (3) culturing the intestinal stem cell-like cells obtained in step (2) to form spheroids; and (4) differentiating the spheroids formed in step (3) to form an intestinal organoid, the step including culture in the presence of a MEK1/2 inhibitor, a DNA methylation inhibitor, a TGF-? receptor inhibitor, and a ?-secretase inhibitor, in addition to an epidermal growth factor, a BMP inhibitor, and a Wnt signal activator.

Abstract: Compositions and methods for binding to a target sequence of interest are provided. The compositions find use in cleaving or modifying a target sequence of interest, visualization of a target sequence of interest, and modifying the expression of a sequence of interest. Compositions comprise RNA-guided nuclease (RGN) polypeptides, CRISPR RNAs, trans-activating CRISPR RNAs, guide RNAs, and nucleic acid molecules encoding the same. Vectors and host cells comprising the nucleic acid molecules are also provided. Further provided are RGN systems for binding a target sequence of interest, wherein the RGN system comprises an RNA-guided nuclease polypeptide and one or more guide RNAs.

Abstract: The present application relates to compositions comprising and methods of using a liposome comprising a pHLIP polypeptide, wherein a lipid bilayer of the liposome is substantially free of the pHLIP polypeptide.

Abstract: Embodiments relate to a modified cell engineered to comprise a modified TCR-CD3 complex, wherein the CD3?, ?-chain, CD3?, and/or CD3? chains of the modified TCR-CD3 complex are linked to one or more co-stimulatory signaling domains.

Abstract: The present invention relates to a cell reprogramming method using a physical stimulation-mediated environmental influx, and more specifically, by subjecting differentiated or non-differentiated cells to physical stimulation which can promote an environmental influx, such as ultrasonic waves, laser or heat shock, without the introduction of a reprogramming-inducing factor or a chemical substance to the differentiated cells, the cells can be reprogrammed with just the imposition of an external environmental influx into pluripotent cells or arbitrary differentiated cells having a different expression type from the differentiated or non-differentiated cells, and as such an inducement has a simple and effective production process, the possibility of an autogenic cell therapy can be made greater.

Abstract: The present invention relates to: recombinant adenoviruses, which have an excellent ability to be introduced into stem cells and can be easily introduced into stem cells even at a low concentration; stem cells comprising the same; a gene delivery composition; and a pharmaceutical composition. Stem cells into which adenoviruses are introduced, of the present invention, have excellent in vivo survivability and a treatment effect on tumor cells, and, when used as an anticancer drug, do not cause problems with respect to in vivo survival, hepatotoxicity and the like of stem cells so as to be particularly usable for systemic administration, and have an excellent anticancer effect even in small doses, thereby being widely usable as an anticancer drug using adenoviruses.

Abstract: Novel adeno-associated virus (AAV) vectors in nucleotide and amino acid forms and uses thereof are provided. The isolates show specific tropism for certain target tissues, such as blood stem cells, liver, heart and joint tissue, and may be used to transduce stem cells for introduction of genes of interest into the target tissues. Certain of the vectors are able to cross tightly controlled biological junctions, such as the blood-brain barrier, which open up additional novel uses and target organs for the vectors, providing for additional methods of gene therapy and drug delivery.

Abstract: The present invention includes compositions and methods for an HLA-A2 specific chimeric antigen receptor (CAR). In certain embodiments the HLA-A2 specific CAR is expressed on a T regulatory cell. In certain embodiments, the HLA-A2 specific CAR protects transplanted tissue from rejection.

Abstract: The disclosure relates, in some aspects, to compositions and methods for enhanced delivery of a transgene to the central nervous system (CNS) of a subject. In some embodiments, the transgene is delivered by recombinant AAV (rAAV). In some embodiments, the method of enhancing transgene delivery comprises administering a blood brain barrier (BBB)-crossing molecule (e.g., K16ApoE) and an rAAV comprising a transgene to a subject.

Abstract: Use of Cistanche deserticola polysaccharide (CDP) in promoting the proliferation and differentiation of female germline stem cells (FGSCs) is provided. Specifically, the addition of CDP in an in vitro cultivation system can promote the proliferation and differentiation of FGSCs, and especially can enhance the in vitro directed differentiation of FGSCs into oocytes, which provides a new research reference for studying the generation of oocytes in vivo and in vitro and also brings a new hope for research on physiological infertility.

Abstract: A Thy1 gene promoter specifically expressed in neurons and a recombinant vector including the Thy1 gene promoter are provided. The Thy1 gene promoter may be utilized to regulate an expression of a target gene in preparation of an animal model similar to a human.

Abstract: A bioactive glass-ceramic composition as defined herein. Also disclosed are methods of making and using the disclosed compositions.