Abstract: This invention provides methods of treating and abrogating spinal muscular atrophy by administering an antioxidant. Additionally, the invention provides a method of protecting the SMN protein by administering an antioxidant.

Abstract: The present invention provides methods for treating neurological diseases and disorders. Compounds that replace or enhance the function of SMN or alleviate or reduce a phenotype of cells with low SMN protein levels are provided. Screening methods and kits for identifying such compounds also are provided.

Abstract: The present invention provides methods for treating neurological diseases and disorders. Compounds that replace or enhance the function of SMN or alleviate or reduce a phenotype of cells with low SMN protein levels are provided. Screening methods and kits for identifying such compounds also are provided.

Abstract: The invention relates to an isolated nucleic acid encoding a eukaryotic Survival of Motor Neuron-Interacting Protein 1 (SIP1), compositions comprising SIP1 and SIP1 and the spinal muscular atrophy (SMA) disease gene product Survival of Motor Neuron protein (SMN), and diagnostic and therapeutic assays directed to SMA. The invention also relates to another protein that specifically interacts with SMN and is a component of gems, designated Gemin3, and the nucleic acid encoding the protein. Additionally, the invention relates to a novel cell line wherein the endogenous SMN genes have been deleted and where an exogenous nucleic acid encoding SMN has been inserted into the cell such that expression of SMN in the cell is under the control of an inducible promoter. This novel cell line provides a stable genetic system for the study of SMA and for the development of SMA therapeutics.

Abstract: The invention relates to an isolated nucleic acid encoding a eukaryotic Survival of Motor Neuron-Interacting Protein 1 (SIP1), compositions comprising SIP1 and SIP1 and the spinal muscular atrophy (SMA) disease gene product Survival of Motor Neuron protein (SMN), and diagnostic and therapeutic assays directed to SMA. The invention also relates to another protein that specifically interacts with SMN and is a component of gems, designated Gemin3, and the nucleic acid encoding the protein. Additionally, the invention relates to a novel cell line wherein the endogenous SMN genes have been deleted and where an exogenous nucleic acid encoding SMN has been inserted into the cell such that expression of SMN in the cell is under the control of an inducible promoter. This novel cell line provides a stable genetic system for the study of SMA and for the development of SMA therapeutics.

Abstract: The present invention includes methods for modulating the expression of a gene or mRNA in a cell using small nuclear RNAs comprising specific sequence and structural features and further comprising a nucleic acid cargo.

Abstract: The present invention includes assays and kits for detecting the assembly of an RNA binding protein-RNA complex and for detecting the activity of an RNA binding protein.

Abstract: The present invention provides methods for treating neurological diseases and disorders. Compounds that replace or enhance the function of SMN or alleviate or reduce a phenotype of cells with low SMN protein levels are provided. Screening methods and kits for identifying such compounds also are provided.

Abstract: The invention relates to an isolated nucleic acid encoding a eukaryotic Survival of Motor Neuron-Interacting Protein 1 (SIP1), compositions comprising SIP1 and SIP1 and the spinal muscular atrophy (SMA) disease gene product Survival of Motor Neuron protein (SMN), and diagnostic and therapeutic assays directed to SMA. The invention also relates to another protein that specifically interacts with SMN and is a component of gems, designated Gemin3, and the nucleic acid encoding the protein. Additionally, the invention relates to a novel cell line wherein the endogenous SMN genes have been deleted and where an exogenous nucleic acid encoding SMN has been inserted into the cell such that expression of SMN in the cell is under the control of an inducible promoter. This novel cell line provides a stable genetic system for the study of SMA and for the development of SMA therapeutics.

Abstract: The invention relates to an isolated nucleic acid encoding a eukaryotic Survival of Motor Neuron-Interacting Protein 1 (SIP1), compositions comprising SIP1 and SIP1 and the spinal muscular atrophy (SMA) disease gene product Survival of Motor Neuron protein (SMN), and diagnostic and therapeutic assays directed to SMA. The invention also relates to another protein that specifically interacts with SMN and is a component of gems, designated Gemin3, and the nucleic acid encoding the protein. Additionally, the invention relates to a novel cell line wherein the endogenous SMN genes have been deleted and where an exogenous nucleic acid encoding SMN has been inserted into the cell such that expression of SMN in the cell is under the control of an inducible promoter. This novel cell line provides a stable genetic system for the study of SMA and for the development of SMA therapeutics.

Abstract: The invention relates to a method of isolating an interacting protein.

Abstract: The present invention relates to substantially pure FXR1 and FXR2 proteins and isolated nucleic acid molecules encoding the same. Recombinant expression vectors comprising nucleic acid sequences that encode FXR1 and FXR2 protein are also provided. Antibodies which bind to an epitope of FXR1 or FXR2, or FMR1 protein are also provided. The present invention also relates to methods of screening individuals for FMR1 deficiency using antibodies or nucleic acid molecules of the invention and pharmaceutical kits for accomplishing the same.

Abstract: A method of promoting annealing of two nucleic acid molecules that contain complementary nucleotide sequences is disclosed. The method comprises the step of contacting the nucleic acid molecules in the presence of hnRNP U protein. A kit for hybridizing two nucleic acid molecules is disclosed. Isolated DNA molecules encoding the hnRNP U protein, expression vectors comprising the DNA molecule, host cells transformed with the expression vector and a method of producing essentially pure hnRNP U protein are disclosed. Essentially pure hnRNP U protein is disclosed. Methods of facilitating intermolecular interactions between two molecules in which at least one molecule is a nucleic acid by contacting the molecules in the presence of a protein, such as hnRNP U protein, which comprises a highly conserved "RGG box" amino acid sequence pattern are disclosed.