HOLMES, Michael C.,RILEY, Brigit E.,WECHSLER, Thomas,ZEITLER, Bryan,ZHANG, Lei
申请号：
AU2018355343
公开号：
AU2018355343A1
申请日:
2018.10.24
申请国别(地区):
AU
年份:
2020
代理人:
摘要：
The present disclosure is in the field of modulation of genes involved in rare diseases including for diagnostics and therapeutics for rare diseases such as Angelman's Syndrome, Facioscapulohumeral Muscular Dystrophy (FHMD), Amyotrophic Lateral Sclerosis (ALS), Frontotemporal dementia (FTD) and Spinal Muscular Atrophy (SMA).
