The present invention provides, for example, a set of two polypeptides exhibiting the nuclease activity with dependence on light or in the presence of a drug, in which an N-terminal side fragment and a C-terminal side fragment of a Cas9 protein are bound to each of two polypeptides which form a dimer with dependence on light or in the presence of a drug.

Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for TTR.

The invention provides novel pharmaceutical compositions and methods for treating newborns in need of enhanced cardiac function, in particular, newborns suffering from cardiomyopathy, or a related disease or condition.

Novel materials and methods useful for expressing heterologous proteins in prokaryotic cells are provided, including prokaryotic cells expressing FkpA and/or Skp.

A biosensor comprises first and second molecular components and is capable of displaying protease activity in response to a binding event mediated by first and second binding partners of the biosensor. The first and second binding partners may bind each other directly or may both bind a target molecule. At least the first molecular component comprises an autoinhibited protease, whereby the binding event switches the protease frora an autoinhibited inactive state to a protease active state. The second molecular component may activate the protease of the first molecular component by binding a cross-binder which releases the autoinhibitor or by cleaving a linker which releases the autoinhibitor. The first and second molecular components may both have autoinhibited proteases which reciprocally activate each other.

The invention concerns a fusion polypeptide comprising several molecules of folding helper polypeptides, comprising one multimerization domain, in particular Skp, and at least one molecule of SlyD or SlpA, wherein no further target polypeptide sequences are fused to said fusion polypeptide. The invention further concerns an immunoassay and the use of said fusion polypeptide in an immunoassay for reduction of interferences or minimizing false positive results or for stabilizing proteinaceous assay reagents. Further the invention concerns a reagent kit for use in an immunoassay comprising said fusion polypeptide.

Provided herein are methods for preventing or treating post-traumatic stress disorder (PTSD) in a subject, the methods including: administering an agent which inhibits formation of a glucocorticoid receptor (GR)-FK506 Binding Protein 51 (FKBP51) complex (GR-FKBP51 complex), or which disrupts already formed GR-FKBP51 complex, to the subject; thereby reducing a level of GR-FKBP51 complex in the subject and preventing or treating the PTSD. Also provided are methods for diagnosing a subject as having, or being at risk of developing, a PTSD, the methods including steps of: measuring a level of a GR-FKBP51 complex in the subject; comparing the measured level to a reference level of a non-PTSD condition; and identifying the subject as having, or being at risk of developing, PTSD where the measured level is elevated relative to the reference level. Agents, compositions, and/or kits for the diagnosis and/or treatment of PTSD are also described.

The present invention is directed to a composition and method which to treat diseases and to enhance a regulated immune response. More particularly, the present invention is drawn to compositions that are based on dendritic cells modified to express an inducible form of a co-stimulatory polypeptide.

The invention is directed to a system comprising a lentivirus vector particle which encodes at least one guide RNA sequence that is complementary to a first DNA sequence in a host cell genome, a Cas9 protein, and optionally a donor nucleic acid molecule comprising a second DNA sequence. The invention also is directed to a method of altering a DNA sequence in a host cell using such a system, where the host cell can be in a human and the altered DNA can be of the human β-globin gene. The invention also is directed to a fusion protein comprising a Cas9 protein and a cyclophilin A (CypA) protein. The invention also is directed to sequences of vectors that can be used in the system and method.

Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for TTR.