Abstract: The present invention provides RNA-based systems that are capable of silencing expression of a gene in a cell. Also provided are pharmaceutical compositions, cells, and kits that include the systems; cultures of primary cells that have been contacted with the systems; use of the systems in methods of studying protein function in one or more cells; and use of the systems in methods of studying interactions between neurons in culture; and use of the systems in a method of studying the function of mRNA. The present invention further provides methods for silencing expression of a gene in a cell, and methods for determining the function of a gene in a cell. Additionally, the present invention provides systems for use in genetic screening, and methods for performing genetic screening.

Abstract: The present invention provides methods for regulating the growth and/or survival of tumor cells and stem cells by modulating the expression or function of ATF5. The present invention also provides methods for promoting or suppressing differentiation of stem/progenitor cells, for producing differentiated cells and for isolating/purifying differentiated cells, including neural cells. Also provided are differentiated cells, cell populations and transgenic animals comprising same and uses of same. The present invention further provides methods for treating nervous tissue degeneration and for identifying an agent for use in treating nervous tissue degeneration. Methods for promoting apoptosis in neoplastic cells and for treating or preventing tumors, and identifying agents for use in treating or preventing tumors are also provided by the present invention. The present invention further provides methods for identifying agents that inhibit ATF5, agents identified by these methods.

Abstract: The present invention provides a membrane-permeable complex for facilitating the delivery of a double-stranded ribonucleic acid molecule into a cell. Specifically, the invention provides a membrane-permeable complex that comprises a double-stranded ribonucleic acid molecule, such as a small interfering RNA, a cell-penetrating peptide, and a covalent bond linking the double-stranded ribonucleic acid to the cell-penetrating peptide. Also provided are methods of using the membrane-permeable complex of the present invention to deliver the double-stranded ribonucleic acid molecule to a cell or to inhibit expression of a gene product by a cell.

Abstract: The present invention provides methods for promoting or suppressing differentiation of neural stem/progenitor cells, for producing differentiated neural cells, and for isolating/purifying differentiated neural cells. Also provided are differentiated neural cells, cell populations and transgenic animals comprising same, and uses of same. The present invention further provides methods for treating nervous tissue degeneration, and for identifying an agent for use in treating nervous tissue degeneration. Additionally, the present invention provides a therapeutic composition, and methods for treating neural tumors using same. The present invention further provides methods for identifying agents which inhibit ATF5, agents identified by these methods, and uses of same. Also provided are methods for diagnosing neural tumors, for assessing the efficacy of therapy to treat neural tumors, and for assessing the prognosis of a subject who has a neural tumor.

Abstract: The present invention discloses a PC12 cell line stably expressing human A53T &agr;-synuclein, wherein a cell of the cell line is characterized by proteasomal dysfunction, dopaminergic dysfunction, lysosomal dysfunction, and increased non-apoptotic cell death, as compared to cells of suitable control cell line not expressing mutant &agr;-synuclein. Also provided are methods of making the cells of the present invention. The unique phenotype associated with the cells described herein provide a cellular model for the study of Parkinson's Disease, as well as for other synucleinopathic neurodegenerative disorders, including, but not limited to, Dementia with Lewy Bodies, Lewy Body Variant of Alzheimer's Disease, Multiple System Atrophy and Hallervorden-Spatz syndrome.

Abstract: The present invention provides a membrane-permeable complex for facilitating the delivery of a double-stranded ribonucleic acid molecule into a cell. Specifically, the invention provides a membrane-permeable complex that comprises a double-stranded ribonucleic acid molecule, such as a small interfering RNA, a cell-penetrating peptide, and a covalent bond linking the double-stranded ribonucleic acid to the cell-penetrating peptide. Also provided are methods of using the membrane-permeable complex of the present invention to deliver the double-stranded ribonucleic acid molecule to a cell or to inhibit expression of a gene product by a cell.

Abstract: Flavopiridol and its salts are used to inhibit neuronal cell damage or neuronal cell death.

Abstract: 6-Cyclohexyl-1-hydroxy-4-methyl-2(1H)-pyridinone, also known as ciclopirox, and its salts such as ciclopirox olamine are used to inhibit neuronal cell damage or neuronal cell death.