Abstract: Disclosed is a method of treating a subject who has a neurological disease. The neurological disease may be associated with altered C9ORF72 protein activity. In one aspect, the method includes a step of administering an effective dose of a PIKFYVE antisense or inhibitory nucleic acid to a subject in need thereof, thereby rescuing the defects associated with altered C9ORF72 protein activity and/or inhibiting the expression of PIKFYVE gene.

Abstract: Disclosed is a method of treating a subject who has a neurological disease. In one aspect, the method includes a step of administering an effective dose of a SYF2 antisense or inhibitory nucleic acid to a subject in need thereof, thereby restoring nuclear localization of TDP-43.

Abstract: The disclosure relates methods for increasing the efficiency of cellular reprogramming of somatic cells and improving the maturity of the resulting cells.

Abstract: Disclosed is a method of treating a subject who has a neurological disease. The neurological disease may be associated with altered C9ORF72 protein activity. In one aspect, the method includes a step of administering an effective dose of a pharmaceutical composition to a subject in need thereof, thereby rescuing the defects associated with altered C9ORF72 protein activity. Also described are methods for identifying a compound for inhibiting motor neuron degeneration.

Abstract: Disclosed is a method of treating a subject who has a neurological disease. The neurological disease may be associated with altered C9ORF72 protein activity. In one aspect, the method includes a step of administering an effective dose of a pharmaceutical composition to a subject in need thereof, thereby rescuing the defects associated with altered C9ORF72 protein activity. Also described are methods for identifying a compound for inhibiting motor neuron degeneration.

Abstract: The present invention provides methods of transdifferentiation of somatic cells, e.g., a fibroblast, into a nociceptor cell, e.g., an induced nociceptor (iNociceptors) with characteristics of a typical nociceptor cell. The present invention also relates to an isolated population comprising iNociceptors, compositions, their use in the study of cellular and molecular mechanisms of peripheral pain generation and peripheral neuropathy, use in in vitro drug discovery assays, pain research, as their use in the treatment of nociceptive pain related diseases or disorders. In particular, the present invention relates to direct conversion of a somatic cell to an iNociceptor cell having nociceptor characteristics by increasing the protein expression of five nociceptor inducing factors selected from Asc11, Myt11, Isl2, Ngn1, Klf7 in a somatic cell, to convert the fibroblast to an iNociceptors which express the markers of adult nociceptors.

Abstract: The present inventions relate to methods and compositions useful for improving the efficiency of inducing the generation of neurons from non-neuronal cell types, for example, by contacting the cell or cell culture medium with one or more agents which inhibit Activin and/or PLK1 signaling. Also disclosed are methods for promoting neuron survival, for example, by inhibiting Activin and/or PLK1 signaling, and methods for promoting the survival of intermediates in a cell differentiation pathway, for example, by inhibiting Activin and/or PLK1 signaling.

Abstract: The disclosure features a method of producing a reprogrammed cell (e.g. an induced pluripotent stem cell or an undifferentiated cell) from a differentiated (e.g. somatic) cell. In some embodiments, the methods includes contacting a differentiated (e.g. somatic cell) with a TGFBR1 inhibitor or anti-TGF-?-antibody to produce a reprogrammed cell (e.g. pluripotent stem cell or undifferentiated cell). Embodiments of the present invention relate to a reprogrammed cell and methods and compositions for producing a chemically produced reprogrammed cell or populations thereof.