The present invention provides for the incorporation of target sequences of microRNAs into the 3UTR region of a gene of interest in nucleic acid vectors. The invention also provides for an expression system comprising such vectors, a pharmaceutical composition comprising such vectors, as well as methods of treating or preventing cardiovascular disease by using such vectors.

The present invention relates to Sor CS1-like agents, including Sor CS1, nucleic acid molecule encoding expression of Sor CS1 and fragments thereof, as well as vectors containing said nucleic acid and to cells expressing Sor CS1 and said fragments, for use in the treatment of insulin resistance.

Several embodiments disclosed herein relate generally to methods and compositions for the generation of biological pacemakers. In some embodiments, the methods comprise contacting non-pacemaker cells with one or more transcription factors (in vivo or in vitro) and inducing pacemaker functionality in the cells.

Provided herein is a method of delivering nucleic acid molecules to a compartmentalized tissue or organ of a subject. Also provided herein are uses and processes for delivering a nucleic acid molecule to the parenchyma of a compartmentalized tissue or organ. The methods and uses can be used in the treatment of diseases and conditions and in industrial, agricultural and veterinary applications. Also provided herein are compositions containing an adenovirus or adeno-associated virus or other recombinant virus formulated for administration to the parenchyma of a tissue or organ.

Provided herein are prime-boost regimens and materials used therein. The prime-boost regimens enhance the immune response to a target antigen. The vaccines used for boost are comprised of recombinant attenuated metabolically active Listeria that encodes an expressible antigen that is cross-reactive with the target antigen. In some examples, the immune response is a cellular immune response.

Compositions and methods for improving cardiac function, myocardial contractility and relaxation in a mammal are provided. Cardiomyocytes transfected with one or more expression vectors comprising a ribonucleotide reductase subunit R1-encoding nucleic acid sequence and a ribonucleotide reductase subunit R2-encoding nucleic acid sequence operably linked to a promoter are grafted to a mammalian myocardium. Also provided are compositions and methods for delivering dATP to a myocardium through grafting of donor cells overexpressing R1 and R2. dATP is thereby produced in situ and delivered through gap junctions established between donor cells and host cardiomyocytes. Alternatively, viral vector(s) having the R1 and R2-encoding construct(s) are administered to the mammal directly. Improvement of cardiac function can also be effected by administration of vectors comprising a nucleic acid sequence encoding a L48Q, 61 Q, or L57Q cTnC variant.

The present disclosure provides compositions for viral gene therapy, e.g. Adeno-Associated virus-directed gene therapy, and methods of using the same for the treatment and/or prevention of cholesterol storage diseases or disorders, such as Niemann-Pick disease, Type C.

The present invention relates to a pharmaceutical composition for preventing or treating a heart failure and a method for screening a therapeutic agent for preventing or treating a heart failure. The pharmaceutical composition of the present disclosure comprises the CCN5 or CCN2CT protein, or a genetic carrier comprising a nucleotide sequence encoding the CCN5 or the CCN2CT protein, exhibiting dramatic prevention or treatment efficacies on a heart failure even without surgical treatments accompanied with transplanting a donor heart.

The present invention provides methods of reprogramming cells, for example, directly reprogramming a somatic cell of a first cell type into a somatic cell of a second cell type, are described herein. In particular, the present invention generally relates to methods for reprogramming a cell of an endoderm origin to a cell having pancreatic -cell characteristics. The present invention also relates to an isolated population comprising reprogrammed cells, compositions and their use in the treatment of diabetes mellitus. In particular, the present invention relates to reprogramming a cell of an endoderm origin to a cell having pancreatic -cell characteristics by increasing the protein expression of at least one transcription factor selected from Pdx1, Ngn3 or MafA in the cell of endoderm origin to reprogram the cell of an endoderm cell to a cell which exhibits at least one or at least two characteristics of an endogenous pancreatic -cell.

Herpes Simplex Virus (HSV) antigens that elicit an HSV-specific immune response and can be used to treat or prevent HSV infection are provided. Nucleic acid sequences, polypeptides, vectors, and compositions, as well as methods to induce an immune response against HSV, treat or prevent HSV disease, induce a T cell response against HSV, and induce an antibody response against HSV also are provided.