Osman, Erkan Y <br/><br/>
Morpholino antisense oligonucleotides targeting intronic repressor Element1 improve phenotype in SMA mouse models.  [electronic resource] 

 -  Human molecular genetics  Sep 2014 
 - 4832-45 p.  digital 
<br/><br/> Publication Type: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't 
<br/><br/><label>ISSN: </label>1460-2083 
<br/><br/><label>Standard No.: </label>10.1093/hmg/ddu198  doi 
<br/><br/><label>Subjects--Topical Terms: </label><br/>Animals<br/>Disease Models, Animal<br/>Genetic Therapy--methods<br/>Humans<br/>Introns<br/>Mice<br/>Morpholinos--administration & dosage<br/>Muscular Atrophy, Spinal--genetics<br/>Regulatory Sequences, Nucleic Acid<br/>Survival Rate<br/>Survival of Motor Neuron 2 Protein--genetics<br/>Weight Gain