Therapeutics that directly increase SMN expression to treat spinal muscular atrophy.
Shababi
Monir
M
Department of Veterinary Pathobiology, University of Missouri, Columbia, MO, USA. shababim@missouri.edu
Mattis
Virginia B
VB
Lorson
Christian L
CL
D000818:Animals; D016503:Drug Delivery Systems; D005091:Exons; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D009134:Muscular Atrophy, Spinal / Q000235:genetics / Q000628:therapy*; D012326:RNA Splicing; D055533:Survival of Motor Neuron 1 Protein / Q000235:genetics / Q000378:metabolism; D055540:Survival of Motor Neuron 2 Protein / Q000235:genetics / Q000378:metabolism
C526471:SMN1 protein, human; C526477:SMN2 protein, human; D055533:Survival of Motor Neuron 1 Protein; D055540:Survival of Motor Neuron 2 Protein