The invention provides recombinant DNA molecules useful for providing efficient expression of proteins in transgenic plants, as well as compositions and methods for using the recombinant DNA molecules. In particular embodiments, the invention provides recombinant DNA molecules and constructs comprising sequences encoding transit peptides and operably linked sequences conferring herbicide tolerance.

The invention relates, in part, to compounds, compositions, and methods useful to treat cancer in cells and subjects. In some aspects the invention includes compounds and methods that increase chemo-sensitivity and/or reduce chemo-resistance in a cancer cell. In certain aspects the invention includes compounds and methods useful to kill cancer cells.

A TAT-FXN fusion polypeptide useful in treating subjects diagnosed with Friedrich's Ataxia, hypertrophic cardiomyopathy, or both are disclosed, as are related methods of treatment and pharmaceutical compositions.

The present invention relates to the preparation of an antibody analogue capable of being activated reversibly, and uses thereof, and provides a fusion protein comprising an inactive first fragment of an antibody analogue is fused to a stimulus-induced dimerization protein.

The specification provides programmable base editors that are capable of introducing a nucleotide change and/or which could alter or modify the nucleotide sequence at a target site in mitochondrial DNA (mtDNA) with high specificity and efficiency. Moreover, the disclosure provides fusion proteins and compositions comprising a programmable DNA binding protein (e.g., a mitoTALE, a mitoZFP, or a CRISPR/Casp) and double-stranded DNA deaminase that is capable of being delivered to the mitochondria and carrying out precise installation of nucleotide changes in the mtDNA. The fusion proteins and compositions are not limited for use with mtDNA, but also may be used for base editing of any double-stranded target DNA.

PC1-CTT polypeptides for the treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD) are provided and can be or include PC1-CTT (SEQ ID NO:1) or a functional fragment or variant thereof. In some embodiments, the PC1-CTT polypeptide is a fusion protein or conjugate further including a functional element such as a protein transduction domain, fusogenic polypeptide, targeting signal, or expression and/or purification tag. Nucleic acids encoding the disclosed PC1-CTT polypeptides and other therapeutic proteins are also provided. In some embodiments, the nucleic acid encodes a TOP or TOP-like motif. The nucleic acids can be RNA or DNA, and can be, for example, a vector such as a plasmid or viral vector, or an mRNA. Methods of treatment are provided and typically include administering a subject in need thereof an effective amount of a disclosed polypeptide or nucleic acid.

This invention provides biosensors, cell models, and methods of their use for monitoring heme and oxygen. Biosensors can include targeting domains, sensing domains and reporting domains. Biosensors can be introduced into cells reprogrammed to represent experimental or pathologic cells of interest. Model cells expressing the biosensors can be contacted with putative bioactive agents to determine possible activities.

This invention provides biosensors, cell models, and methods of their use for monitoring heme and oxygen. Biosensors can include targeting domains, sensing domains and reporting domains. Biosensors can be introduced into cells reprogrammed to represent experimental or pathologic cells of interest. Model cells expressing the biosensors can be contacted with putative bioactive agents to determine possible activities.

The present invention relates to methods and compositions for the prevention or treatment of neurodegenerative diseases.

Compositions and methods of treatments of cells are provided for altering the phenotype of a cell by administering an oligonucleotide complex to the cell, the complex having two strands and chemical modifications.