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1. (WO2016059166) HUMANIZED NEUROMELANIN-CONTAINING RODENT

Pub. No.:    WO/2016/059166    International Application No.:    PCT/EP2015/073909
Publication Date: Fri Apr 22 01:59:59 CEST 2016 International Filing Date: Fri Oct 16 01:59:59 CEST 2015
IPC: A01K 67/027
C12N 9/02
A61K 49/00
Applicants: FUNDACIÓ HOSPITAL UNIVERSITARI VALL D'HEBRON - INSTITUT DE RECERCA
INSTITUCIÓ CATALANA DE RECERCA I ESTUDIS AVANÇATS
CENTRO DE INVESTIGACIÓN BIOMÉDICA EN RED ENFERMEDADES NEURODEGENERATIVAS (CIBERNED)
Inventors: VILA BOVER, Miquel
CARBALLO CARBAJAL, Iria
BOVÉ BADELL, Jordi
CUADROS ARASA, Thais
LAGUNA TUSET, Ariadna
Title: HUMANIZED NEUROMELANIN-CONTAINING RODENT
Abstract:
The invention provides a humanized rodent exhibiting age-dependent production and intracellular accumulation of human-like neuromelanin, up to levels reached in elder humans, within Parkinson's disease-vulnerable neuronal groups. The humanized neuromelanin-containing rodent stably expresses the human tyrosinase gene in at least the dopaminergic neurons of the substantia nigra pars compacta and can be a wild-type rodent to which the human tyrosinase gene has been transduced via a viral vector, or a transgenic rodent that expresses the human gene in a constitutive or inducible form. In any of these cases, the rodent develops features of Parkinson's disease such as nigro-striatal dopaminergic neurodegeneration and motor impairment, making it a valuable tool for the (i) search of biomarkers for the early detection, diagnosis or progression of the disease, and (ii) the screening of new therapies with disease-modifying potential to combat PD.