Brian K. Kaspar,Kevin Foust,KASPAR, Brian, K.,FOUST, Kevin
申请号：
US13270840
公开号：
US20120177605A1
申请日:
2011.10.11
申请国别(地区):
US
年份:
2012
代理人:
摘要：
The present invention relates to methods and materials useful for systemically delivering polynucleotides to the spinal cord. Use of the methods and materials is indicated, for example, for treatment of lower motor neuron diseases such as spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS) as well as Pompe disease and lysosomal storage disorders.
