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COMPOSITIONS AND METHODS FOR TREATMENT OF NEUROGENERATIVE DISEASES
专利权人:
Alexander C. Flynn
发明人:
Alexander C. Flynn
申请号:
US15089174
公开号:
US20170035860A1
申请日:
2016.04.01
申请国别(地区):
US
年份:
2017
代理人:
摘要:
Medical compositions and methods of treating or preventing neurodegeneration in a human suffering from or that is at risk of or susceptible to neurodegeneration or cellular dysfunction associated with expression or impaired cellular function of a neuronal protein encoded by one or more genes that code for alpha-synuclein (SNCA), Parkin RBR E3 ubiquitin protein ligase, (PARK2), Leucine-rich repeat kinase 2 (LRRK2), PTEN-induced putative kinase/(PINK1), Daisuke-Junko 1, (DJ-1) and ATPase type 13A2 (ATP13A2), are disclosed. Methods of treatment for these disorders is also provided, comprising administering a vector into a cell, wherein the vector facilitates expression of a molecular component that alters one of the aforementioned genes in the cell or expression of the gene in the cell, the gene being implicated in an etiology of the neurological deficit.
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