Pathophysiology associated with a single gene (mass1)...

C - Chemistry – Metallurgy – 12 – Q

Patent

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C12Q 1/00 (2006.01) A01K 67/027 (2006.01) C12N 5/06 (2006.01) C12N 15/90 (2006.01)

Patent

CA 2523537

A novel gene is provided which is associated with audiogenic seizures in mice. The disclosed gene is known as the Monogenic Audiogenic Seizure-susceptible gene or massl, with a product designated MASS 1. Nucleic acid molecules that encode for MASS I have been identified and purified. Mammalian genes encoding MASSI are also provided. The invention also provides recombinant vectors comprising nucleic acid molecules that code for a MASS I protein. The invention also relates to transgenic animals in which one or both alleles of the endogenous mass1 gene is mutated. The invention further relates to a hearing impairment associated with the Frings MASS1 mutation. More specifically, the invention characterizes a moderate and non-progressive hearing impairment which leads to the development of audiogenic seizures.

L'invention concerne un nouveau gène qui est associé aux crises audiogènes chez les souris. Ce gène est connu sous le nom de mass 1 (Monogenic Audiogenic Seizure-susceptible gene), et son produit est désigné par MASS 1. Les molécules d'acide nucléique codant pour MASS1 ont été identifiées et purifiées. Des gènes de mammifères codant pour MASS 1 sont également décrits. L'invention concerne en outre des vecteurs recombinés comprenant les molécules d'acide nucléique codant pour une protéine MASS1. L'invention se rapporte par ailleurs à des animaux transgéniques porteurs d'une mutation sur un ou deux allèles du gène mass 1 endogène, ainsi qu'à une déficience auditive associée à la mutation de MASS 1 chez les Frings. De manière plus spécifique, cette invention caractérise une déficience auditive modérée non progressive conduisant à l'apparition de crises audiogènes.

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