Composition and methods for the therapeutic use of an atonal-associated sequence for deafness, osteoarthritis and abnormal cell proliferation
AssigneeBaylor College of Medicine
InventorsHuda Y. Zoghbi, Hugo Bellen, Nessan Bermingham, Bassem Hassan, Nissim Ben-arie
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Abstract
Compositions and methods are disclosed for the therapeutic use of an atonal-associated nucleic acid or amino acid sequence. Also, an animal heterozygous for an atonal-associated gene inactivation is also disclosed having at least one atonal-associated nucleic acid sequence replaced by insertion of a heterologous nucleic acid sequence used to detect expression driven by an atonal-associated promoter sequence, wherein the inactivation of the atonal-associated nucleic acid sequence prevents expression of the atonal-associated gene.
Claims8
A method of treating animal for imbalance disorder, comprising delivering directly to a cell in an inner ear of said animal a therapeutically effective amount of an atonal-associated amino acid molecule or nucleic acid molecule, wherein said amino acid molecule has hair cell generating activity and the sequence of said amino acid molecule has at least about 80% identity to SEQ ID NO:58 (Hath1) and comprises at least about 80% identity to SEQ ID NO:70, and wherein said nucleic acid molecule encodes said amino acid molecule, and wherein upon said delivery step the imbalance disorder is treated.
Dependencies are inferred from the claim text.