Abstract: Disclosed herein are compounds and methods for decreasing PrP and preventing, ameliorating, or treating a prion disease or conformational neurodegenerative disorder, in an individual in need thereof. Examples of disease conditions that can be ameliorated with the administration of antisense compounds targeted to PrP include Creutzfeldt-Jakob disease (CJD); variant Creutzfeldt-Jakob Disease (vCJD); Gerstmann-Straussler-Scheinker syndrome; fatal familial insomnia; kuru; Bovine Spongiform Encephalopathy (BSE), e.g. “mad cow disease”; Chronic Wasting Disease (CWD); scrapie; transmissible mink encephalopathy; feline spongiform encephalopathy; ungulate spongiform encephalopathy; Alzheimer's disease; Parkinson's disease; Huntington's disease; and Amyotrophic Lateral Sclerosis (ALS).

Abstract: Provided herein are methods, compounds, and compositions for reducing expression of huntingtin mRNA and protein in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate Huntington's disease, or a symptom thereof.

Abstract: Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.

Abstract: Disclosed herein are compounds and methods for decreasing PrP and preventing, ameliorating, or treating a prion disease or conformational neurodegenerative disorder, in an individual in need thereof. Examples of disease conditions that can be ameliorated with the administration of antisense compounds targeted to PrP include Creutzfeldt-Jakob disease (CJD); variant Creutzfeldt-Jakob Disease (vCJD); Gerstmann-Straussler-Scheinker syndrome; fatal familial insomnia; kuru; Bovine Spongiform Encephalopathy (BSE), e.g. “mad cow disease”; Chronic Wasting Disease (CWD); scrapie; transmissible mink encephalopathy; feline spongiform encephalopathy; ungulate spongiform encephalopathy; Alzheimer's disease; Parkinson's disease; Huntington's disease; and Amyotrophic Lateral Sclerosis (ALS).

Abstract: The subject invention provides a method for producing an olfactory GPCR in a cell. In general, the methods involve introducing an expression cassette containing a promoter operably linked to a nucleic acid encoding an olfactory PCR into a macroglial cell, e.g., a Schwann or oligodendritic cell, and maintaining the cell under conditions suitable for production of the olfactory GPCR. Also provided is a macroglial cell containing a recombinant nucleic acid encoding an olfactory GPCR, methods of screening for modulators of olfactory GPCR activity, and a kit for producing an olfactory GPCR in a macroglial cell. The invention finds most use in research on flavors and fragrances, and, consequently, has a variety of research and industrial applications.

Abstract: Disclosed herein are compounds, compositions and methods for modulating the expression of huntingtin in a cell, tissue or animal. Further provided are methods of slowing or preventing Huntington's disease progression using an antisense compound targeted to huntingtin. Additionally provided are methods of delaying or preventing the onset of Huntingtin's disease in an individual susceptible to Huntingtin's Disease. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders.

Abstract: The subject invention provides a method for producing an olfactory GPCR in a cell. In general, the methods involve introducing an expression cassette containing a promoter operably linked to a nucleic acid encoding an olfactory PCR into a macroglial cell, e.g., a Schwann or oligodendritic cell, and maintaining the cell under conditions suitable for production of the olfactory GPCR. Also provided is a macroglial cell containing a recombinant nucleic acid encoding an olfactory GPCR, methods of screening for modulators of olfactory GPCR activity, and a kit for producing an olfactory GPCR in a macroglial cell. The invention finds most use in research on flavors and fragrances, and, consequently, has a variety of research and industrial applications.

Abstract: In a preferred embodiment, the present invention relates to a Schwannoma cell line derived from a Schwann tumor in a patient with Neurofibromatosis type 2 (NF2) and immortalized with HPV E6-E7 genes. The cell line has a unique splice site mutation of the NF2 gene. The immortalized cell line is non-tumorigenic but has altered growth properties such as higher proliferation rate and independence of Schwann cell growth factors. Methods of using this unique cell line for pharmacologic screening are disclosed.

Abstract: In a preferred embodiment, the present invention relates to a Schwannoma cell line derived from a Schwann tumor in a patient with Neurofibromatosis type 2 (NF2) and immortalized with HPV E6-E7 genes. The cell line has a unique splice site mutation of the NF2 gene. The immortalized cell line is non-tumorigenic but has altered growth properties such as higher proliferation rate and independence of Schwann cell growth factors. Methods of using this unique cell line for pharmacologic screening are disclosed.

Abstract: The subject invention provides a method for producing an olfactory GPCR in a cell. In general, the methods involve introducing an expression cassette containing a promoter operably linked to a nucleic acid encoding an olfactory PCR into a macroglial cell, e.g., a Schwann or oligodendritic cell, and maintaining the cell under conditions suitable for production of the olfactory GPCR. Also provided is a macroglial cell containing a recombinant nucleic acid encoding an olfactory GPCR, methods of screening for modulators of olfactory GPCR activity, and a kit for producing an olfactory GPCR in a macroglial cell. The invention finds most use in research on flavors and fragrances, and, consequently, has a variety of research and industrial applications.

Abstract: Disclosed herein are compounds, compositions and methods for modulating the expression of huntingtin in a cell, tissue or animal. Further provided are methods of slowing or preventing Huntington's disease progression using an antisense compound targeted to huntingtin. Additionally provided are methods of delaying or preventing the onset of Huntingtin's disease in an individual susceptible to Huntingtin's Disease. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders.