Abstract: A method of modulating some or all copies of a gene in a cell is provided including introducing into a cell one or more ribonucleic acid (RNA) sequences that comprise a portion that is complementary to all or a portion of each of the one or more target nucleic acid sequences, and a nucleic acid sequence that encodes a Cas protein and maintaining the cells under conditions in which the Cas protein is expressed and the Cas protein binds and modulates the one or more target nucleic acid sequences in the cell.

Abstract: The present invention relates to the area of in vitro cell populations useful for generating vascular networks in vitro and are suitable for use in vivo for regeneration of vascular tissue. In some embodiments, the bipotent cell population of the present invention comprise endothelial cells and pericytes that express vascular endothelial cadherin and are 95% or more positive for CD105 and CD146, and which work syergistically to recreate vascular tissues in vitro.

Abstract: The present invention provides methods and devices for isolating cells from a subject by circulating the subject's body fluid over an affinity moeity coupled matrix to isolate cells from a subject either ex vivo or in vivo. One aspect of the invention is directed to connecting a subject to a system capable of circulating the subject's body fluid through an affinity moiety coupled matrix, such that the affinity moiety coupled matrix is capable of binding to and extracting target cells from the body fluid, and then eluting the target cells from the affinity moiety. Another aspect of the invention is directed to the apparatus for isolating cells from a subject, comprising a blood circulation system with an arterial side blood circuit for extracting blood and flowing the blood over an affinity moiety coupled matrix that binds to and extracts target cells and a venous side blood circuit for returning the blood to the patient.

Abstract: An implantable medical product and method of use for substantially reducing or eliminating harsh biological responses associated with conventionally implanted medical devices, including inflammation, infection and thrombogenesis, when implanted in in a body of a warm blooded mammal. The bioremodelable pouch structure is configured and sized to receive, encase and retain an electrical medical device therein and to allow such device to be inserted into the internal region or cavity of the pouch structure; with the pouch structure formed from either: (a) first and second sheets, or (b) a single sheet having first and second sheet portions. After receiving the electrical device, the edges around the opening are closed by suturing or stapling. The medical device encased by the bioremodelable pouch structure effectively improves biological functions by promoting tissue regeneration, modulated healing of adjacent tissue or growth of new tissue when implanted in the body of the mammal.

Abstract: A genome-replication-deficient and transcription/competent negative/strand RNA virus, which can be used for the expression of transgenes and in particular for the area of vaccine development. In particular, a recombinant Sendia virus containing a viral genome with a mutation in the gene P, which leads to loss of capacity for replication without loss of capacity for secondary transcription, and at least one sequence coding for a heterologous gene product. Furthermore, a nucleocapsid of the Sendai virus, a genome of the Sendai virus, a DNA molecule that codes for the genome and/or antigenome of the Sendai virus. One aspect is a pharmaceutical composition containing the Sendai virus, the nucleocapsid, or the viral genome as active substance, and a medical use thereof, especially as a vaccine.

Abstract: A CpG-modified recombinant adeno-associated viral (AAV) vector is described. The vector carries a nucleic acid molecule comprising AAV inverted terminal repeat (ITR) sequences and an exogenous gene sequence under the control of regulatory sequences which control expression of the gene product, in which the nucleic acid sequences carried by the vector are modified to significantly reduce CpG di-nucleotides such that an immune response to the vector is reduced as compared to the unmodified AAV vector. Also provided are methods and regimens for delivering transgenes using these AAV viral vectors, in which the innate immune response to the vector and/or transgene is significantly modulated.

Abstract: Genetically modified cells that are compatible with multiple subjects, e.g., universal donor cells, and methods of generating said genetic modified cells are provided herein. The universal donor cells comprise at least one genetic modification within or near at least one gene that encodes one or more MHC-I or MHC-II human leukocyte antigens or component or transcriptional regulator of the MHC-I or MHC-II complex, at least one genetic modification that increases the expression of at least one polynucleotide that encodes a tolerogenic factor, and optionally at least one genetic modification that increases or decreases the expression of at least one gene that encodes a survival factor.

Abstract: Genetically modified cells that are compatible with multiple subjects, e.g., universal donor cells, and methods of generating the genetic modified cells are provided herein. The universal donor cells comprise at least one genetic modification within or near at least one gene that encodes one or more MHC-I or MHC-II human leukocyte antigens or component or transcriptional regulator of the MHC-I or MHC-II complex, at least one genetic modification that increases the expression of at least one polynucleotide that encodes a tolerogenic factor, and optionally at least one genetic modification that increases or decreases the expression of at least one gene that encodes a survival factor.

Abstract: Double stranded mRNA (ds mRNA), e.g., produced in vitro, where one strand encodes a protein of interest and the other strand is hydrogen bonded to at least a portion of the coding region for the protein, as well as methods of making and using the ds mRNA, are provided.

Abstract: The present invention provides a method for producing a ? myosin heavy chain in cardiac muscle cells differentiated from induced pluripotent stem cells derived from Homo sapiens. In the present method, first, a liquid culture medium containing the cardiac muscle cells is supplied onto a substrate comprising a first electrode, a second electrode and insulative fibers on the surface thereof. At least a part of the insulative fibers is located between the first electrode and the second electrode in a top view of the substrate. Then, the substrate is left at rest. Finally, the cardiac muscle cells are cultivated, while a pulse electric current is applied to the cardiac muscle cells through the first electrode and the second electrode.

Abstract: The invention provides genetically modified non-human animals that express a humanized MHC II protein (humanized MHC II ? and ? polypeptides), as well as embryos, cells, and tissues comprising the same. Also provided are constructs for making said genetically modified animals and methods of making the same. Methods of using the genetically modified animals to study various aspects of human immune system are provided.

Abstract: The present disclosure relates to recombinant adeno-associated virus (rAAV) delivery of a GALGT2 polynucleotide. The disclosure provides rAAV and methods of using the rAAV for GALGT2 gene therapy of neuromuscular disorders. Exemplary neuromuscular disorders include, but are not limited to, muscular dystrophies such as Duchenne muscular dystrophy, Congenital Muscular Dystrophy 1A and Limb Girdle Muscular Dystrophy 2D.

Abstract: The present disclosure relates to genetically modified non-human animals (e.g., genetically-modified mice) that express a human or chimeric (e.g., humanized) CD27. The present disclosure also relates to methods of generating the genetically-modified animals (e.g., genetically modified mice), and methods of using the genetically modified non-human animals (e.g., genetically modified mice) described herein.

Abstract: NK cell based cancer immunotherapy, and particularly genetically modified NK92 cell-based immunotherapy is enhanced by expression CXCL12 and/or by suppression or deletion of CXCR4 in the natural killer cells to so reduce aggregation, rejection, and/or fratricide of the natural killer cells. Provided herein are genetically engineered NK (natural killer) cell comprising a recombinant nucleic acid encoding at least a portion of chemokine C—X—C motif ligand 12 (CXCL12), and a transcript for downregulation of chemokine C—X—C motif receptor 4 (CXCR4).

Abstract: Disclosed are cell membrane-derived nanovesicles, a method of preparing the nanovesicles, and a pharmaceutical composition and a diagnostic kit using the nanovesicles. The cell membrane-derived nanovesicles may prevent potential adverse effects because intracellular materials (e.g., genetic materials and cytosolic proteins) unnecessary for delivering therapeutic or diagnostic substances are removed from the nanovesicles. In addition, as the nanovesicles may be targeted to specific cells or tissues, therapeutic or diagnostic substances may be predominantly delivered to the targeted cells or tissues, while delivery of the substances may be inhibited. Therefore, the nanovesicles may alleviate suffering and inconvenience of patients by reducing adverse effects of therapeutic substances and by improving efficacy of the substances.

Abstract: A method for creating an animal model of traumatic optic nerve injury, including fully exposing an internal segment of an optic canal as well as adjacent anterior skull base, posterior ethmoid sinus and lateral sphenoid sinus walls through an ethmoid sinus-sphenoid sinus operation pathway under an endoscope, and impacting different sites of the internal segment of the optic canal with controllable impact force to cause optic nerve injury so as to prepare a controllable and quantifiable TONI bionic elastic injury animal model reflecting contusion to an internal segment of an optic canal in a human TONI clinical injury state. With less intracranial combined injury to the animal, the survival rate is high. Different sites of the optic canal are impacted with quantifiable elastic force for the quantitative and qualitative purposes with respect to the injured parts and the injury degree.

Abstract: A method of modulating some or all copies of a gene in a cell is provided including introducing into a cell one or more ribonucleic acid (RNA) sequences that comprise a portion that is complementary to all or a portion of each of the one or more target nucleic acid sequences, and a nucleic acid sequence that encodes a Cas protein and maintaining the cells under conditions in which the Cas protein is expressed and the Cas protein binds and modulates the one or more target nucleic acid sequences in the cell.

Abstract: This disclosure relates to genetically modified animal expressing human or chimeric (e.g., humanized) programmed death-ligand 1 (PD-L1, PDL1, or B7-H1), and methods of use thereof. In one aspect, the disclosure relates to genetically-modified, non-human animals whose genome comprises at least one chromosome comprising a sequence encoding a human or chimeric programmed death-ligand 1 (PD-L1).

Abstract: A method useful for identifying and isolating live circulating tumor cells is described. The method utilizes an adenoviral vector comprising a replication-competent adenovirus in which the E1 gene region is expressed under the control of a telomerase-specific promoter and further comprises a second expression cassette containing a marker protein, optionally fused to a detectable cell surface marker to permit detection of circulating tumor cells lacking cell surface markers. The method involves combining ex vivo a test sample from a patient suspected of having circulating tumor cells, an adenoviral probe system, and culture media for the cells. The test sample is incubated with the adenoviral system for a sufficient time to permit expression of the reporter protein. The marker gene expression can thereafter be quantitated and the marker-expressing cells may optionally be collected for further analysis.

Abstract: A method for predicting an effect of a medication or a treatment regimen to a subject suffering from a cancer, the method comprises: (A) obtaining a tissue from the subject; (B) dissociating the tissue to obtain a multicellular cluster, wherein the multicellular cluster comprises the cancer cell; (C) culturing the multicellular cluster on a cellulose sponge; (D) exposing the cultured multicellular cluster to the medication or the treatment regimen; and (E) measuring a first survival rate of the cancer cell before exposing to the medication or the treatment regimen and a second survival rate of the cancer cell after exposing to the medication or the treatment regimen, when the second survival rate is lower than the first survival rate, the method predicts positive effect of the medication or the treatment regimen to the subject.