Palhais, Bruno <br/><br/>
Splice-shifting oligonucleotide (SSO) mediated blocking of an exonic splicing enhancer (ESE) created by the prevalent c.903+469T>C MTRR mutation corrects splicing and restores enzyme activity in patient cells.  [electronic resource] 

 -  Nucleic acids research  May 2015 
 - 4627-39 p.  digital 
<br/><br/> Publication Type: Journal Article; Research Support, Non-U.S. Gov't 
<br/><br/><label>ISSN: </label>1362-4962 
<br/><br/><label>Standard No.: </label>10.1093/nar/gkv275  doi 
<br/><br/><label>Subjects--Topical Terms: </label><br/>Anemia, Megaloblastic--enzymology<br/>Cell Line<br/>Cells, Cultured<br/>Exons<br/>Ferredoxin-NADP Reductase--genetics<br/>HEK293 Cells<br/>Homocystinuria--enzymology<br/>Humans<br/>Mutation<br/>Oligonucleotides<br/>RNA Splice Sites<br/>RNA Splicing<br/>Regulatory Sequences, Ribonucleic Acid