Compositions and methods for the therapeutic use of an...

C - Chemistry – Metallurgy – 12 – N

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C12N 15/09 (2006.01) A61K 38/17 (2006.01) C07K 14/435 (2006.01) C07K 14/46 (2006.01) C07K 14/465 (2006.01) C07K 14/47 (2006.01) C12N 15/12 (2006.01) C12N 15/63 (2006.01) C12N 15/67 (2006.01) C12N 15/85 (2006.01) C12Q 1/68 (2006.01) A61K 38/00 (2006.01) A61K 48/00 (2006.01)

Patent

CA 2375106

Compositions and methods are disclosed for the therapeutic use of an <i>atonal</i>-associated nucleic acid or amino acid sequence. Also, an animal heterozygous for an <i>atonal</i>-associated gene inactivation is also disclosed having at least one <i>atonal</i>-associated nucleic acid sequence replaced by insertion of a heterologous nucleic acid sequence used to detect expression driven by an <i>atonal</i>-associated promoter sequence, wherein the inactivation of the <i>atonal</i>-associated nucleic acid sequence prevents expression of the <i>atonal</i>-associated nucleic acid.

L'invention se rapporte à des compositions et à des procédés permettant une utilisation thérapeutique d'une séquence d'acides aminés ou d'acides nucléiques associée au gène <i>atonal</i>. Elle se ra pporte également à un animal hétérozygote qui est destiné à l'inactivation de gènes associés à <i>atonal</i> et dont au moins une séquence d'acides nucléiques associée à <i>atonal</i> a été remplacée par insertion d'une séquence d'acides nucléiques hétérologues dans le but de déceler l'expression commandée par une séquence promotrice associée à <i>atonal</i>, l'inactivation de la séquence d'acides nucléiques associée à <i>atonal</i> empêchant l'expression de l'acide nucléique associé à <i>atonal</i>.

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