| 摘要 |
The present invention features compositions and methods for transformation of cells of the inner ear and treatment of conditions of the inner ear using such methods. More specifically, cells of an inner ear of a subject are genetically altered to operatively incorporate a nucleotide sequence which expresses a gene product of interest (e.g., a therapeutic gene product). Preferably, the inner ear cell into which the DNA of interest is introduced and expressed is a cell of the cochlea, more preferably a cell of the spiral ligament, spiral limbus, stria vascularis, organ of Corti, spiral ganglion, and/or Reissner's membrane, and/or an auditory hair cell. The DNA of interest, preferably present within an adeno-associated viral vector, is introduced through a cannula inserted in the round or oval window and in communication with the perilymph or endolymph. Preferably, introduction of the DNA of interest is accomplished using an osmotic minipump. |
