THE TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK
发明人:
Brian D. McCabe,Livio Pellizzoni
申请号：
US14434501
公开号：
US20150258170A1
申请日:
2013.10.10
申请国别(地区):
US
年份:
2015
代理人:
摘要：
The present invention provides for methods for diagnosing and treating a motor neuron disease. More specifically, the present invention offers new methods for diagnosing and treating SMA or SMN deficiencies and monitoring treatment. It is possible to identify a subject having a symptom of the disease, and then administer to the subject a therapeutically effective amount of one or more proteins or a gene delivery vehicle or pharmaceutical composition comprising one or more genes selected from the group consisting of Transmembrane protein 41B (Stasimon), Chromosome 19 open reading frame 54 (Rashomon), Tetraspanin 31, Poly (ADP-ribose) polymerase family member 1, Histidyl-tRNA synthetase-like, Chloride channel 7, and Nucleolar protein 1.
