Targeted retrograde gene delivery to motor neurons

Details Targeted retrograde gene delivery to motor neurons Targeted retrograde gene delivery to motor neurons

C - Chemistry, Metallurgy

12

N

C12N 15/86 (2006.01) A61K 31/7088 (2006.01) A61K 48/00 (2006.01) A61P 25/28 (2006.01) C12N 15/864 (2006.01) C12N 5/0793 (2010.01)

Patent

CA 2471812

Methods are disclosed for delivering a heterologous gene to a cell body of a neuron by contacting either the synaptic portion of a neuron, for example or a muscle tissue innervated by the neuron, with a viral vector comprising a heterologous gene, wherein the viral vector enters said neuron and is retrogradely moved to the cell body. Additionally, methods for expressing secreted proteins from a nerve cell body as well as methods for treating neurodegenerative disorders are described.

L'invention concerne des méthodes permettant de transférer un gène hétérologue sur un corps cellulaire d'un neurone par contact soit avec la partie synaptique d'un neurone, par exemple, soit avec un tissu musculaire innervé par ledit neurone, un vecteur viral comprenant un gène hétérologue. Ce vecteur viral pénètre dans ledit neurone, puis il est rétrogradé vers le corps cellulaire. L'invention concerne également des méthodes permettant d'exprimer des protéines sécrétées d'un corps cellulaire nerveux ainsi que des méthodes permettant de traiter des maladies neurodégénératives.

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