Abstract: Compositions of, methods of making, and methods of using hybrid nanoparticles comprise at least one semiconducting polymer and at least one nonsemiconducting polymer. Compositions of, methods of making and methods of using hybrid nanoparticles comprise at least one semiconducting polymer and non-semiconducting polymers wherein the non-semiconducting polymer comprises more than one non-semiconducting polymer such that at least one non-semiconducting polymer is functionalized for bioconjugation. The hybrid nanoparticles are polarization-sensitive and have low mass ratios with large fluorescence.

Abstract: The disclosure in some aspects relates to recombinant adeno-associated viruses having distinct tissue targeting capabilities. In some aspects, the disclosure relates to gene transfer methods using the recombinant adeno-associated viruses. In some aspects, the disclosure relates to isolated AAV capsid proteins and isolated nucleic acids encoding the same.

Abstract: Provided are a cell line stably expressing voltage-gated sodium channel (Nav1.5), wherein a SCN5A gene (NCBI accession no. NM_198056) encoding Nav1.5 and a puromycin selection marker are inserted into a chromosome of a host cell, and a method of measuring cardiotoxicity of a test substance comprising: (1) contacting a test substance to the cell line of claim 1; and (2) measuring magnitude of voltage-gated sodium channel (Nav1.5) in the cell line in contact with the test substance.

Abstract: Provided herein are neuroprotective molecules containing a sequence of 25-35 contiguous nucleotides between nucleotide 1 and nucleotide 50 of a mature human tRNA and at least four contiguous guanosine-containing nucleotides, where the sequence of 25-35 contiguous nucleotides contains a D-loop stem structure, the at least four contiguous guanosine-containing nucleotides are located at the 5? end of the neuroprotective molecule, and the neuroprotective molecule contains at least one deoxyribonucleotide. Also provided are neuroprotective molecules containing a sequence of 25-35 contiguous between nucleotide 1 and nucleotide 50 of a mature human tRNACYS; and at least four contiguous guanosine-containing nucleotides, where the sequence of 25-35 contiguous nucleotides contains a D-loop stem structure and the at least four contiguous guanosine-containing nucleotides are located at the 5? end of the neuroprotective molecule.

Abstract: The present invention relates to a viral vector for expression of the cell death-inducing PUMA protein, for use in gene therapy, for example for the treatment of rheumatoid arthritis. This vector consists in particular of a recombinant adenovirus expressing the gene encoding the PUMA protein and of a recombinant baculovirus containing a coxsackie-adenovirus receptor (CAR).

Abstract: A trifunctional molecule comprising a target-specific ligand, a ligand that binds a protein associated with the TCR complex and a T cell receptor signaling domain polypeptide is provided. Engineering T cells with this novel receptor engenders antigen specific activation of numerous T cell functions, including cytokine production, degranulation and cytolysis.

Abstract: Provided are methods, systems, and kits for cell processing, e.g., for therapeutic use, such as for adoptive cell therapy. The provided methods include transduction methods, in which cells and virus are incubated under conditions that result in transduction of the cells with a viral vector. The incubation in some embodiments is carried out in an internal cavity of a generally rigid centrifugal chamber, such as a cylindrical chamber made of hard plastic, the cavity of which may have a variable volume. The methods include other processing steps, including those carried out in such a chamber, including washing, selection, isolation, culture, and formulation. In particular, the disclosure relates to method providing advantages over available processing methods, such as available methods for large-scale processing. Such advantages include, for example, reduced cost, streamlining, increased efficacy, increased safety, and increased reproducibility among different subjects and conditions.

Abstract: Improved methods of culturing embryos in media having amounts of lactate that have not previously been recognized as beneficial for embryo development. Also, compositions, devices and kits related to the same.

Abstract: Some embodiments of the invention comprise methods, systems, and compositions to selectively induce, whether in vitro or in vivo, the neuronal differentiation of multipotent stromal cells through the application of microRNAs, including but not limited to miRNA-124, miRNA-137 and/or miRNA-9* expression products of those miRNAs, and molecules and compositions containing functional elements of those miRNAs. Some embodiments of the invention also comprise the therapeutic administration and use of such induced cells to treat mammalian injuries and diseases, including but not limited to, nervous system injuries or diseases that may otherwise result in decreased cell or system function.

Abstract: Methods for increasing specificity of RNA-guided genome editing, e.g., editing using CRISPR/Cas9 systems, using truncated guide RNAs (tru-gRNAs).

Abstract: Correlations between polymorphisms and breast cancer are provided. Methods of diagnosing, prognosing, and treating breast cancer are provided. Systems and kits for diagnosis, prognosis and treatment of breast cancer are provided. Methods of identifying breast cancer modulators are also described.

Abstract: A self-regulating gene expression construct comprises a single promoter in operative association with a repressor sequence (e.g., bacterial repressor lacI or gaiR), operator sequence(s) responsive to the expressed repressor protein, and a transgene. A dual-regulating construct comprises a single promoter controlling expression of a bacterial repressor sequence and a transgene, and which, in the presence of a first inducer molecule, transcribes the transgene and repressor; and a ribozyme in association with an aptamer sequence, the aptamer sequence capable of interacting with a second inducer molecule to terminate mRNA degradation by the ribozyme. Also provided are recombinant vectors or viruses containing the self-regulating or dual self-regulating constructs and cells containing the vectors. Such compositions are useful in methods of treating a diseases using gene therapy.

Abstract: The present disclosure provides compositions and methods that rapidly and selectively modify cells of the immune system to achieve therapeutic objectives. The methods can be practiced in vivo and any cell type that expresses a known marker can be targeted for a therapeutic objective.

Abstract: The invention provides improved compositions for adoptive T cell therapies for B cell related conditions.

Abstract: A method for detecting a mutation in an amino acid at position 93 of a hepatitis C virus NS5A protein, the method including: synthesizing cDNA using, as a template, hepatitis C virus RNA in a sample; and performing a real-time PCR with a cycling probe method using, as a template, the cDNA; wherein a primer set used in the real-time PCR is a certain primer set; and wherein probes used in the real-time PCR include certain probes.

Abstract: The present invention relates to a method for generating batches of lymphocytes with averaged potency. In particular, the present invention relates to a method of pooling lymphocytes from different donors to avoid NK alloreactivity and anti-HLA immune response. Lymphocytes from each donor are inactivated for at least a gene encoding a TCR component, and are pooled together before be administrated to a subject in need thereof. Thus, this method allows generating batches of lymphocytes with averaged potency, particularly to treat cancer, viral infection or auto-immune disease. The present invention also relates to a batch of lymphocytes obtainable by this method. The batch of lymphocytes can be used to be administrated to one or several patients, being made available as an “off the shelf” therapeutic product, in particular to treat cancer, auto-immune disease or viral infection.

Abstract: The subject matter of the present invention is in particular the use of an amino acid sequence of IDE, or of an analogue or fragment thereof, or of at least one nucleic acid sequence encoding this sequence, as a biomarker, or as an active agent, with regard to a dandruff condition of the scalp.

Abstract: The present disclosure provides a controlled release composition comprising a plurality of microparticles and a matrix, wherein: the plurality of microparticles comprises a first material; the matrix comprises a second material; and the melting temperature of the first material is higher than the melting temperature of the second material. Also provided are methods of making and using the same.

Abstract: Disclosed are capsid-modified rAAV particles and expression vectors, as well as compositions and pharmaceutical formulations that comprise them. Also disclosed are methods of preparing and using novel capsid-protein-mutated particle or rAAV vector constructs in a variety of diagnostic and therapeutic applications including, inter alia, as delivery agents for diagnosis, treatment, or amelioration of one or more diseases, disorders, or dysfunctions of the mammalian eye. Also disclosed are methods for subretinal delivery of therapeutic gene constructs to mammalian photoreceptors and retinal pigment epithelial cells, as well as use of the disclosed compositions in the manufacture of medicaments for a variety of in vitro and/or in vivo applications including the treatment of a variety of inherited retinal diseases.

Abstract: Disclosed are nanoparticles that are introduced into cells and express a specific protein and a manufacturing method thereof. More particularly, the present invention relates to mRNA nanoparticles, which increase the expression of a specific protein capable of stimulating the cellular immune system to induce cellular immune responses and are thus applicable to treat a variety of diseases, do not require passage across the nuclear envelope because a desired gene is delivered not as plasmid DNA itself but in the form of mRNA, thus improving the efficiency of protein expression, and the nanoparticles are generated through a one-step process with a relatively small amount of plasmid DNA via rolling circle transcription (RCT), thereby providing a simple and economical process for gene delivery. The present invention is also concerned with such mRNA nanoparticles.