Additional file 1 of Preclinical evaluation of AAV9-coSMN1 gene therapy for spinal muscular atrophy: efficacy and safety in mouse models and non-human primates
This dataset includes raw electropherogram files (FSA format) generated by Multiplex Ligation-dependent Probe Amplification (MLPA) analysis of SMN1 and SMN2 genes in three affected members (Patients 7
Spinal muscular atrophy (SMA) is a common genetic motor neuron (MN) disease caused by low levels of the ubiquitously expressed housekeeping survival motor neuron (SMN) protein, whereas concomitant ove
Neurotransmission defects and motoneuron degeneration are hallmarks of Spinal Muscular Atrophy, a monogenetic disease caused by the deficiency of the SMN protein. In the present study, we show that sy
Spinal and bulbar muscular atrophy (SBMA) is a neuromuscular disorder caused by a polyglutamine expansion in the androgen receptor (AR). Previous studies have shown that transcriptional dysregulation