ISS-N1 makes the first FDA-approved drug for spinal muscular atrophy
EW Ottesen - Translational neuroscience, 2017 - degruyter.com
Spinal muscular atrophy (SMA) is one of the leading genetic diseases of children and
infants. SMA is caused by deletions or mutations of Survival Motor Neuron 1 (SMN1) gene …
infants. SMA is caused by deletions or mutations of Survival Motor Neuron 1 (SMN1) gene …
Oligonucleotide‐induced alternative splicing of serotonin 2C receptor reduces food intake
Z Zhang, M Shen, PJ Gresch… - EMBO molecular …, 2016 - embopress.org
The serotonin 2C receptor regulates food uptake, and its activity is regulated by alternative
pre‐mRNA splicing. Alternative exon skip** is predicted to generate a truncated receptor …
pre‐mRNA splicing. Alternative exon skip** is predicted to generate a truncated receptor …