Lateral flow devices, methods and kits for performing lateral flow western blot assays are provided.

Described herein are compositions and methods for the immobilization of passenger molecules in a dense matrix of ADP-ribose within the vault particle. The present disclosure also describes a method for altering the physicomechanical properties (e.g. density, compressive strength, electrostatic properties, etc.) of packaged vaults for enhanced stability and/or downstream functionality. In addition, the present disclosure also describes compositions and methods for altering amino acid sequence of the vault protein in the vault particle by amino acid mutation, amino acid insertion and/or amino acid deletion to package passenger molecules and/or to target vault particles to specific receptors or ligands.

A lentiviral vector system for expressing a lentiviral particle is disclosed. The lentiviral vector system includes a therapeutic vector, an envelope plasmid, and at least one helper plasmid. The lentiviral vector system can produce a lentiviral particle for inhibiting PARP expression in neuron cells of a subject afflicted with Parkinson's disease.

Compounds for treatment of diseases having acidic or hypoxic diseased tissues and pharmaceutical compositions comprising the compounds, as well as methods for making and using the compounds and compositions.

The present invention relates to methods of treating a condition associated with mTORC1 hyperactivation or TSC2-deficient cancer, the method comprising administering to a subject having the cancer a pharmaceutically-effective amount of a poly(ADP-ribose) polymerase 1 (PARP1) inhibitor. In some embodiments, the condition associated with mTORC1 hyperactivation is tuberous sclerosis complex (TSC). In some embodiments, the condition associated with mTORC1 hyperactivation is lymphangioleiomyomatosis (LAM). In some embodiments, the condition associated with mTORC1 hyperactivation is TSC2-deficient cancer.

The present invention is directed to a method, kit, system and fusion protein for detecting binding to an ADP-ribosyl group or a polymer thereof, wherein said group or polymer is coupled to a peptide or protein, the method comprising the steps of: i) providing a first entity comprising a first label or tag, said entity comprising an amino acid sequence comprising a cysteine residue whereto at least one ADP-ribosyl group or an analog thereof is coupled via an S-glycosidic bond; ii) contacting in an assay said first entity with a second entity, said second entity being or suspected of being capable of binding to an ADP-ribosyl group or polymer thereof coupled to a peptide or protein; and iii) measuring a signal derived from said first label or localized by said tag, wherein the signal detected is different or is localized differently when said second entity binds to said at least one ADP-ribosyl group of the first entity from the signal detected when the binding interaction between said second entity and said ADP-ribosyl group has not occurred. The kit of the present invention provides means to perform the method of the invention.

Provided herein are split reporter systems for detecting poly-ADP ribose polymerase activity in living systems. In some aspects, the split reporter systems comprise a first fusion protein comprising a first fragment of a reporter protein functionally linked to a first poly-ADP ribose binding moiety; and a second fusion protein comprising a second fragment of the reporter protein functionally linked to a second poly-ADP ribose binding moiety wherein the first and second fragments of the reporter protein are each non-functional and capable of recombining, optionally in the presence of a substrate, to form a functional reporter protein capable of producing a detectable signal. Also provided are methods of use thereof.

The invention relates to compositions of vault complexes for use as adjuvants for stimulating a cellular immune response to an antigen, for example a tumor antigen, and methods of using the vault complexes in the treatment of diseases, such as cancer.

The present invention relates to a method for determining sensitivity to a simultaneous inhibitor against poly ADP ribose polymerase (PARP) and Tankyrase. According to the present invention, a colorectal treatment effect can be maximized by sorting patients having sensitivity to the simultaneous inhibitor against PARP and Tankyrase.

The invention provides methods and compositions for enhancing the efficacy of cancer therapies through modulation of BAL1 and/or BBAP. Also provided are methods for predicting the efficacy of cancer therapies or treating cancer in a subject through modulation of BAL1 and/or BBAP. Further provided are methods for identifying compounds that are capable of modulating BAL1-BBAP complexes.