Abstract: The present disclosure relates to wound treatment and therapy and the promotion of tissue regeneration following injury. In particular, it relates to a microRNA-146a and nanoceria conjugate for improving wound healing and, in some embodiments, preventing adverse ventricular remodeling following myocardial infarction.

Abstract: The present disclosure is directed to methods and compositions for inhibiting a cancer cell using nucleic acid sequences encoding elephant p53 or elephant p53 amino acid sequences.

Abstract: The present invention relates to a novel library for the generation of muteins and to novel muteins derived from human lipocalin 2 (Lcn2, hNGAL) and related proteins that bind a given target with detectable affinity. The invention also relates to corresponding nucleic acid molecules encoding such a mutein and to a method for their generation. The invention further relates to a method for producing such a mutein. For example, such muteins may serve to bind and deplete pathological forms of natural biomolecules such as the amyloid beta peptide in Alzheimer's disease or may target the fibronectin extra-domain B, which is associated with tumor neovasculature.

Abstract: Disclosed herein is a recombinant adeno-associated virus (AAV) vector comprising (a) a variant AAV2 capsid protein, wherein the variant AAV2 capsid protein comprises at least four amino acid substitutions with respect to a wild type AAV2 capsid protein; wherein the at least four amino acid substitutions are present at the following positions in an AAV2 capsid protein sequence: 457, 492, 499 and 533; and (b) a heterologous nucleic acid comprising a nucleotide sequence encoding a gene product.

Abstract: RNA encoding an immunogen is delivered to a large mammal at a dose of between 2 ?g and 100 ?g. Thus the invention provides a method of raising an immune response in a large mammal, comprising administering to the mammal a dose of between 2 ?g and 100 ?g of immunogen-encoding RNA. Similarly, RNA encoding an immunogen can be delivered to a large mammal at a dose of 3 ng/kg to 150 ng/kg. The delivered RNA can elicit an immune response in the large mammal.

Abstract: A method for treating a patient suffering from a neuronal hypo-kinetic disease or a neuronal hyper-kinetic disease by modulating neuronal activity in the: internal globus pallidus (GPi), in the anterior motor thalamus and/or in the external globus pallidum (GPe) and/or in the subthalamic nucleus (STN) by utilizing suppressor and/or enhancer DREADDs is provided.

Abstract: The present technology relates to compositions and methods for modulating expression of genes which include a target oligonucleotide sequence, such as repeats of a particular oligonucleotide sequence containing 3 to 10 nucleotides. In particular aspects, the present technology relates to agents having a formula A-L-B, wherein -L- is a linker; A- is a Brd4 binding moiety; and —B is a nucleic acid binding moiety, such as a polyamide or complementary oligonucleotide, that specifically binds to the target oligonucleotide sequence.

Abstract: Disclosed herein are high transducing replication defective herpes simplex virus (HSV) vectors of McKrae strain.

Abstract: This invention relates to a transgenic vector for transducing cells of a mammal's CNS and transgenically expressing a protein in the mammal's CNS. The transgenic vector comprises a virus-derived vector, a nucleic acid sequence encoding the protein, and an endogenous ATP1A3 promoter sequence. This invention also relates to a composition comprising a recombinant AAV vector comprising a nucleic acid sequence encoding a ATP1A3 protein, in a form compatible with administration into the CNS. The invention also relates to a method for treating a subject having a neurological disorder associated with mutations in the ATP1A3 gene and a method for delivering a transgenic ATP1A3 DNA to the central nervous system of a mammal by administering the recombinant AAV vector into the mammal's CNS.

Abstract: The present invention is directed to new compositions that are described for the simultaneous, controlled dose delivery of a variety of biomolecules into phagocytic cells.

Abstract: RNA encoding an immunogen is delivered to a large mammal at a dose of between 2 ?g and 100 ?g. Thus the invention provides a method of raising an immune response in a large mammal, comprising administering to the mammal a dose of between 2 ?g and 100 ?g of immunogen-encoding RNA. Similarly, RNA encoding an immunogen can be delivered to a large mammal at a dose of 3 ng/kg to 150 ng/kg. The delivered RNA can elicit an immune response in the large mammal.

Abstract: Chimeric antigen receptors containing human CD19 antigen binding domains are disclosed. Nucleic acids, recombinant expression vectors, host cells, antigen binding fragments, and pharmaceutical compositions, relating to the chimeric antigen receptors are also disclosed. Methods of treating or preventing cancer in a subject, and methods of making chimeric antigen receptor T cells are also disclosed.

Abstract: An object of the present invention is to provide novel mesenchymal stem cells demonstrating superior therapeutic effects against various diseases, a novel pharmaceutical composition containing these mesenchymal stem cells, and a method for preparing the same. The present invention relates to ROR1-positive mesenchymal stem cells. The ROR1-positive mesenchymal stem cells are preferably positive for CD29, CD73, CD90, CD105 and CD166 and are derived from umbilical cord or adipose tissue.

Abstract: A nucleic acid carrier according to an embodiment of the present disclosure includes CpG-ODN-RNA conjugate and a porous silica particle carrying the conjugate inside pores thereof. In this regard, the nucleic acid carrier of the present invention can stably deliver loaded nucleic acid molecules to a body and release the same to a target, thereby increasing Type 1 interferon and exhibiting RNA-inherent functions.

Abstract: Disclosed herein is a formulation comprising an extracellular vesicle (EV), and a therapeutic active agent induced or embedded in the EV. According to preferred embodiments of the present disclosure, the EV is isolated from umbilical cord mesenchymal stem cells, and the active agent may be a growth factor, an immune-modulating agent, a small molecule, an siRNA, cDNA or a plant ingredient; for example, curcumin. Also disclosed herein are methods for producing the present formulation, and uses of the present formulation in the treatment of various diseases.

Abstract: Described herein are lipid nanoparticles comprising cationic lipids and other lipids and also comprising engineered nucleases facilitate transfer of nucleic acids to cells.

Abstract: The present disclosure provides RNA-guided CRISPR-Cas effector proteins, nucleic acids encoding same, and compositions comprising same. The present disclosure provides ribonucleoprotein complexes comprising: an RNA-guided CRISPR-Cas effector protein of the present disclosure; and a guide RNA. The present disclosure provides methods of modifying a target nucleic acid, using an RNA-guided CRISPR-Cas effector protein of the present disclosure and a guide RNA. The present disclosure provides methods of modulating transcription of a target nucleic acid.

Abstract: Provided herein are compounds of Formula (I), and salts thereof, wherein each instance of RL is independently optionally substituted C6-C40 alkenyl. Further provided are compositions comprising a compound of Formula (I) and an agent. Further provided are methods and kits using the compositions for delivering an agent to a subject or cell and for treating and/or preventing a range of diseases. Further provided are methods of preparing compounds of Formula (I) and precursors thereof.

Abstract: Methods and composition for modulating a target genome and stable integration of a transgene of interest into the genome of a cell are disclosed.

Abstract: A method of manufacturing a multilayered cell sheet of neural crest stem cells (NCSCs), includes: (1) isolating and culturing NCSCs from peripheral nerves; (2) embedding the cultured NCSCs in a hydrogel; (3) culturing the hydrogel comprising the NCSCs embedded therein under stressed culture conditions in which a physical support is applied; and (4) culturing the resulting hydrogel of step (3) under non-stressed culture conditions in which a physical support is removed.