COMPOSITIONS AND METHODS FOR MODULATING SMN ACTIVITY

摘要

The present invention provides novel genetic modifiers of SMN function. In particular, the present invention provides methods of treating spinal muscular atrophy (SMA) in a subject in need thereof by manipulating specific genes in the BMP and FGF signaling cascades as well as genes related to control of translation and the cytoskeleton. Hypomorphic Smn Drosophila strains, which more closely mimic (SMA) pathology, and methods of using such strains for screening putative therapeutic compounds are also disclosed.