Abstract: The invention provides HDAC inhibitors that may be used as therapeutics for the treatment of a neurodegenerative or neuromuscular condition. The invention provides compounds of formula I: The invention also provides pharmaceutical compositions and articles of manufacture that include these compounds, as well as methods of treating and methods of preventing or delaying the onset of a neurodegenerative or neuromuscular condition.

Abstract: The invention provides polyamides which bind genes having expanded oligonucleotide repeat sequences, which binding modulates transcription. The invention further provides methods of modulation of the transcription of such genes, and the use of polyamides as therapeutic agents to treat diseases associated with such genes.

Abstract: Methods are provided for decreasing the rate or inhibiting neoplastic cell proliferation by reducing or inhibiting histone H4 gene expression or histone H4 activity. Also provided are methods of treating a patient with a neoplastic disease, and compositions useful for treating a cancer patient, including, for example, a composition containing small interfering RNA molecules that reduce or inhibit histone H4 expression in a cell or a composition containing a pyrrole-imidazole polyamide operatively linked to a chemotherapeutic molecule.

Abstract: Zinc finger proteins of the Cys2His2 type represent a class of malleable DNA binding proteins which may be selected to bind diverse sequences. Typically, zinc finger proteins containing three zinc finger domains, like the murine transcription factor Zif268 and the human transcription factor Spl, bind nine contiguous base pairs (bp). To create a class of proteins which would be generally applicable to target unique sites within complex genomes, the present invention provides a polypeptide linker that fuses two three-finger proteins. Two six-fingered proteins were created and demonstrated to bind 18 contiguous bp of DNA in a sequence specific fashion. Expression of these proteins as fusions to activation or repression domains allows transcription to be specifically up or down modulated within cells. Polydactyl zinc finger proteins are broadly applicable as genome-specific transcriptional switches in gene therapy strategies and the development of novel transgenic plants and animals.