Pyk2-based gene therapy attenuates cognitive deficits associated to huntington’s disease

In the present invention it is shows that the inactivation of the Pyk2 gene does not alter hippocampal development but prevents hippocampal-dependent memory tasks and LTP. Inventors clearly provide evidence for multiple roles of Pyk2 in spine morphology and post synaptic structure.
Thus, the inventors used direct overexpression of PYK2 by AAV-mediated gene transfer into the brain of Huntington’s mouse model and found that overexpression of PYK2 in this model improves synaptic plasticity and spine density deficits wich is also accompanied by a rescue of spatial memory.
Accordingly it was demonstrate that PYK2 may restore cognitive functions in neurodegenerative diseases. Thus the present invention relates to a method of treating neurodegenerative disease in a subject in need thereof comprising administering to the subject a therapeutically effective amount of a vector which comprises a nucleic acid molecule encoding for PYK2 polypeptide.

Keywords: neurodegenerative disease - cognitif deficits
Patent Application number: European Procedure (Patents) (EPA) - 24 Mars 2017 - 17305340.6
Inventors:
GIRAULT Jean-Antoine,BRITO Veronica Ines,GINES Silvia,GIRALT Albert
Publications:
Nat Commun. 2017 May 30;8:15592. doi: 10.1038
comms15592.

Reference:

BIO17059-T1

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Patent filling date: 24-03-2017
Rare disease: Yes
Second indication: No

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