The present invention provides polynucleotide vectors for high expression of heterologous genes. Some vectors further comprise novel transposons and transposases that further improve expression. Further disclosed are vectors that can be used in a gene transfer system for stably introducing nucleic acids into the DNA of a cell. The gene transfer systems can be used in methods, for example, gene expression, bioprocessing, gene therapy, insertional mutagenesis, or gene discovery.

The present invention provides polynucleotide vectors for high expression of heterologous genes. Some vectors further comprise novel transposons and transposases that further improve expression. Further disclosed are vectors that can be used in a gene transfer system for stably introducing nucleic acids into the DNA of a cell. The gene transfer systems can be used in methods, for example, gene expression, bioprocessing, gene therapy, insertional mutagenesis, or gene discovery.

In alternative embodiments, provided are compositions, including products of manufacture and kits, and methods, for remotely-controlled and non-invasive manipulation of intracellular nucleic acid expression, genetic processes, function and activity in live cells, e.g., adding functions or changing or adding specificities for immune cells, for monitoring physiologic processes, for the correction of pathological processes and for control of therapeutic outcomes. In alternative embodiments, provided are ultrasound-based thermal or mechanical stimulations, and thermo- or mechano-sensitive protein, either synthetically engineered or natively (endogenously) occurring, integrated to control the production of intracellular nucleic acid and gene expression, e.g., for the expression of biological-active proteins, which can be used, in alternative embodiments, for diagnostic or therapeutic purposes. In alternative embodiments, exemplary thermo- and mechanogenetic systems provided herein allow a deep penetration of stimulation and manipulation in vivo at centimeter-level depth with high spatiotemporal precision.

The present invention relates to a gene therapy vector which is useful in the treatment or prevention of hypertrophic cardiomyopathy in a subject in need thereof. The gene therapy vector of the invention comprises a nucleic acid sequence encoding a cardiac sarcomeric protein and a cardiomyocyte-specific promoter which is operably linked to said nucleic acid sequence. The invention furthermore relates to a cell which comprises the gene therapy vector. Pharmaceutical compositions which comprise the gene therapy vector and/or a cell comprising said vector are also provided. In another aspect, the invention relates to a method for treating or preventing hypertrophic cardiomyopathy in a subject by introducing the gene therapy vector of the invention into a subject in need of treatment.

The present invention relates to a gene therapy vector which is useful in the treatment or prevention of hypertrophic cardiomyopathy in a subject in need thereof. The gene therapy vector of the invention comprises a nucleic acid sequence encoding a cardiac sarcomeric protein and a cardiomyocyte-specific promoter which is operably linked to said nucleic acid sequence. The invention furthermore relates to a cell which comprises the gene therapy vector. Pharmaceutical compositions which comprise the gene therapy vector and/or a cell comprising said vector are also provided. In another aspect, the invention relates to a method for treating or preventing hypertrophic cardiomyopathy in a subject by introducing the gene therapy vector of the invention into a subject in need of treatment.

A fertility restoration gene in wheat and uses thereof, belonging to the field of plant biotechnology, specifically relating to the cloning of a recessive nuclear male sterility gene and a promoter thereof, and uses thereof in hybrid breeding. The fertility restoration gene FRG1 was successfully cloned by flow cytometry and high-throughput sequencing. The FRG1 gene can completely restore the male fertility of a Lanzhou genic male sterile mutant or allelic mutants thereof, which lays a foundation for the construction of a new wheat hybrid breeding technology system, meanwhile, which provides more possibilities to solve the technical bottlenecks of three lines and two lines hybridization technology of wheat, such as unstable fertility of sterile lines, limited resources for hybrid variety, the complexity of seed production technology and the high cost of seed production. The provided gene and method for propagation and maintenance of the sterile line have important significance and application value for hybrid breeding in wheat.

The present invention provides polynucleotide vectors for high expression of heterologous genes. Some vectors further comprise novel transposons and transposases that further improve expression. Further disclosed are vectors that can be used in a gene transfer system for stably introducing nucleic acids into the DNA of a cell. The gene transfer systems can be used in methods, for example, gene expression, bioprocessing, gene therapy, insertional mutagenesis, or gene discovery.

A method to prevent, inhibit or treat one or more symptoms associated with a disease of the central nervous system by intrathecally, intracerebroventricularly or endovascularly administering a rAAV encoding a gene product associated with the disease, e.g., a mammal in which the gene product is absent or present at a reduced level relative to a mammal without the disease.

A chimeric opsin GPCR protein comprising a light-sensitive upstream opsin portion and a target GPCR portion comprising a chimeric CT is provided that expresses strongly and is targeted into the correct subcellular compartment of target cells. The chimeric opsin GPCR protein activates efficiently the native G-protein specific to the target GPCR pathway eliciting a physiological response comparable to the native target GPCR. Nucleic acid molecules encoding the chimeric opsin GPCR protein as well as a capsids, vectors, cells and carriers comprising or expressing the chimeric opsin GPCR protein are also provided. Furthermore, a method of genetically engineering a chimeric opsin GPCR protein and medical applications of the chimeric opsin GPCR protein are provided.

The present invention provides polynucleotide vectors for high expression of heterologous genes. Some vectors further comprise novel transposons and transposases that further improve expression. Further disclosed are vectors that can be used in a gene transfer system for stably introducing nucleic acids into the DNA of a cell. The gene transfer systems can be used in methods, for example, gene expression, bioprocessing, gene therapy, insertional mutagenesis, or gene discovery.