Abstract: The present invention relates to compounds useful as promoters of the SMN2 gene. The invention also provides pharmaceutically acceptable compositions comprising said compounds and methods of using the compositions in the treatment of Spinal Muscular Atrophy.

Abstract: The modulator compounds described herein modulate muscarinic receptors and are useful for treating muscarinic receptor mediated diseases.

Abstract: The present invention relates to modulators of muscarinic receptors. The present invention also provides compositions comprising such modulators, and methods therewith for treating muscarinic receptor mediated diseases.

Abstract: This invention provides an optical probe useful as an optical probe or sensor of post translational type modifications, such as phosphorylation. The invention comprises a polypeptide moiety, which contains a recognition motif for a post translational type activity and a protease site, which is coupled to a probe moiety. Modification of the polypeptide, by the post translational type activity, results in a modulation of the rate at which a protease cleaves the polypeptide which is sensed by a measurable change in at least one optical property of the optical probe upon cleavage. The present invention also includes a recombinant nucleic acid molecule that encodes an optical probe and a vector and host cell or library of cells that include the recombinant nucleic acid molecule. The optical probe can be used in methods to determine whether a sample, including a cell or a sample from an organism, contains a post-translational type modification activity.

Abstract: The invention provides a compound, useful as an optical probe or sensor of the activity of at least one cytochrome P450 enzyme, and methods of using the compound to screen candidate drugs, and candidate drugs identified by these methods.

Abstract: The invention provides methods of using an optical molecular probe or sensor to screen candidate drugs for their interaction with at least one cytochrome P450 enzyme.

Abstract: The invention provides methods of using a compound as an optical probe or sensor of the activity of at least one cytochrome P450 enzyme, and methods of using the compound to screen candidate drugs, and kits for performing these methods.

Abstract: The invention provides a compound, useful as an optical probe or sensor of the activity of at least one cytochrome P450 enzyme, and methods of using the compound to screen candidate drugs, and candidate drugs identified by these methods.

Abstract: This invention provides an optical probe useful as an optical probe or sensor of post translational type modifications, such as phosphorylation. The invention comprises a polypeptide moiety, which contains a recognition motif for a post translational type activity and a protease site, which is coupled to a probe moiety. Modification of the polypeptide, by the post translational type activity, results in a modulation of the rate at which a protease cleaves the polypeptide which is sensed by a measurable change in at least one optical property of the optical probe upon cleavage. The present invention also includes a recombinant nucleic acid molecule that encodes an optical probe and a vector and host cell or library of cells that include the recombinant nucleic acid molecule. The optical probe can be used in methods to determine whether a sample, including a cell or a sample from an organism, contains a post-translational type modification activity. Such methods can also be used to.

Abstract: The invention provides methods of using a compound as an optical probe or sensor of the activity of at least one cytochrome P450 enzyme, and methods of using the compound to screen candidate drugs, and kits for performing these methods.

Abstract: The invention provides a compound, useful as an optical probe or sensor of the activity of at least one cytochrome P450 enzyme, and methods of using the compound to screen candidate drugs, and candidate drugs identified by these methods.

Abstract: The invention provides a compound, useful as an optical probe or sensor of the activity of at least one cytochrome P450 enzyme, and methods of using the compound to screen candidate drugs, and candidate drugs identified by these methods.

Abstract: This invention provides an optical probe useful as an optical probe or sensor of post translational type modifications, such as phosphorylation. The invention comprises a polypeptide moiety, which contains a recognition motif for a post translational type activity and a protease site, which is coupled to a probe moiety. Modification of the polypeptide, by the post translational type activity, results in a modulation of the rate at which a protease cleaves the polypeptide which is sensed by a measurable change in at least one optical property of the optical probe upon cleavage. The present invention also includes a recombinant nucleic acid molecule that encodes an optical probe and a vector and host cell or library of cells that include the recombinant nucleic acid molecule. The optical probe can be used in methods to determine whether a sample, including a cell or a sample from an organism, contains a post-translational type modification activity.

Abstract: The invention provides a compound, useful as an optical probe or sensor of the activity of at least one cytochrome P450 enzyme, and methods of using the compound to screen candidate drugs, and candidate drugs identified by these methods. The optical probe of the invention is a compound having the generic structure Y-L-Q, wherein Y is selected from the group consisting of Q as herein defined, saturated C.sub.1 -C.sub.20 alkyl, unsaturated C.sub.1 -C.sub.20 alkenyl, unsaturated C.sub.1 -C.sub.20 alkynyl, substituted saturated C.sub.1 -C.sub.20 alkyl, substituted unsaturated C.sub.1 -C.sub.20 alkenyl, substituted unsaturated C.sub.1 -C.sub.20 alkynyl, C.sub.1 -C.sub.20 cycloalkyl, C.sub.1 -C.sub.20 cycloalkenyl, substituted saturated C.sub.1 -C.sub.20 cycloalkyl, substituted unsaturated C.sub.1 -C.sub.20 cycloalkenyl, aryl, substituted aryl, heteroaryl and substituted heteroaryl; L is selected from the group of (--O(substituted ortho-phenyl)CR.sup.2 H).sub.p --, (--O(substituted meta-phenyl)CR.sup.2 H).sub.

Abstract: Neuroprotective drugs are disclosed with at least 3 branches extending outwardly from a center atom or group, each branch having a guanidino group at its terminus. All branches preferably should be identical, and distributed around the center atom or group in a radial manner. Three branches can be bonded to a nitrogen atom, or four branches can be coupled to a carbon atom; other center groups include stable aromatic, cycloalkyl, heterocyclic, or bicyclic structures. Starting reagents are disclosed with a center atom or group, and with reactive groups (such as primary amines or hydroxyl groups) at the ends of short "spacer chains" bonded to the center atom or group. Reagents derived from arginine (an amino acid having a terminal guanidino group) can be bonded to these center components, using protective groups on the arginyl reagents to ensure desired final products with accessible guanidino groups at the ends of spacer chains.

Abstract: Chemical derivatives of nitric oxide are provided which are stable indefinitely in oxygen-containing solutions until photolysis, whereupon they release NO. These compounds have the general formulaA--N.sup.+ (O.sup.-)=N--O--B (I)wherein A is typically a nitrogen- or oxygen-containing substituent and B is a group lablie to photolysis. The compounds are stable and inert in oxygenated aqueous solutions, but release NO upon illumination. Given the ease with which the intensity, timing and location of illumination may be controlled, these compounds are particularly useful in investigating the biological effects of NO with much higher spatial or temporal resolution than heretofore possible.