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Project TitleCompositions and Methods for Diagnosing and Treating Nucleotidde Repeat Distorders
Track CodeFOFF-NRD
Websitewww.lvg.virginia.edu
Short Description

Inventors:
Erin P. Foff, M.D., Benjamin W. Purow, M.D.

Abstract

UVA investigators  have discovered a novel mechanism of disease across a spectrum of fatal neurodegenerative conditions, including a specific and common form of familial ALS.  They have in vitro evidence for miR dysfunction in the presence of nucleotide repeats, and identified at least one putative miR target that itself is disrupted as a consequence.  This target, NETO1, is differentially expressed in the brains of C9+ ALS patients, consistent with their in vitro findings, and experimental manipulation of both repeats and miR-762 effect changes in NETO1. Finally, they have identified at least 2 therapeutic compounds, in use in other conditions, which can lead to miR-762 increases in our cell lines, and thus represent possible novel therapeutics for C9+ disease. 

Licensing Contact:
Patrick Klepcyk
Director, Licensing
klepcyk@virginia.edu
(434) 982-1610

 
Tagschronic disease treatment, in-vitro, brain, therapeutics
 
Posted DateFeb 22, 2017 3:10 PM

Files

File Name Description
Pitch Deck None Download
U.S. 2016 0340732 None Download