Aav mediated gene delivery to cochlear cells

A - Human Necessities – 01 – N

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A01N 63/00 (2006.01) A01N 65/00 (2006.01)

Patent

CA 2571159

The present invention is directed to a method of transducing mammalian cochlear cells, more preferably, cochlear hair cells and support cells. The method involves the delivery of adeno-associated virus (AAV) to a target mammalian cochlear cell. The AAV comprises DNA which is exogenous to the AAV and a promoter operatively linked to the DNA. Preferably, the promoter is a cell specific promoter, e.g., hair cell or support cell specific promoter, and the AAV is serotype 1, 2, 6, or a mixture of two or more serotypes. The present invention also relates to compositions comprising modified AAV useful in transducing specific cochlear cells.

La présente invention concerne un procédé de transduction de cellules cochléaires mammaliennes, de préférence, de cellules cochléaires capillaires et de cellules support. Le procédé comprend la diffusion d'un virus adéno-associé (AAV) à destination d'une cellule cochléaire mammalienne cible. AAV comprend un ADN qui est exogène à AAV et un promoteur lié de façon fonctionnelle à l'ADN. De préférence, le promoteur est un promoteur spécifique à la cellule, p.ex., à la cellule capillaire, ou un promoteur spécifique à la cellule support, et l'AAV est un sérotype 1, 2, 6, ou un mélange de deux ou plusieurs sépotypes. La présente invention concerne aussi des compositions comprenant un AAV modifié utile dans la transduction de cellules cochléaires spécifiques.

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