Animal model, methods for making and using thereof, and composition for treating ataxia
AssigneeCity University of Hong Kong
InventorsSungchil YANG, Eric Hong Jian WONG
Life of this patent
Estimating the term…
Abstract
The present invention provides a platform for in vitro or in vivo study of the correlation between a Purkinje cell-specific, circadian clock gene and ataxia, in particular, a non-human transgenic animal model induced by genetic modification to knockdown the circadian clock gene, Bmal1, which causes abnormal diurnality and loss of certain motor skills and learning ability in a subject. The present invention also relates to methods of making and using the platform for various applications. A composition including a vector carrying the Bmal1 gene for restoring expression thereof in the subject's cerebellum to potentially treat ataxia arising from the Bmal1 gene deficiency is also provided.
Claims20
A non-human animal model having a Purkinje cell-specific gene deficiency leading to ataxia, the Purkinje cell-specific gene being one of the circadian clock genes relating to regulation of circadian rhythm, motor skills and learning ability of a subject.
A composition comprising a Purkinje cell-specific gene relating to regulation of circadian rhythm, motor skills and learning ability of a subject, the Purkinje cell-specific gene being one of the circadian clock genes and expressed in a higher level at daytime than nighttime.
Dependencies are inferred from the claim text.