Malerba, Alberto <br/><br/>
Established PABPN1 intranuclear inclusions in OPMD muscle can be efficiently reversed by AAV-mediated knockdown and replacement of mutant expanded PABPN1.  [electronic resource] 

 -  Human molecular genetics  10 2019 
 - 3301-3308 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/ddz167  doi 
<br/><br/><label>Subjects--Topical Terms: </label><br/>Animals<br/>Dependovirus--genetics<br/>Disease Models, Animal<br/>Gene Knockdown Techniques<br/>Genetic Vectors<br/>Humans<br/>Intranuclear Inclusion Bodies--metabolism<br/>Mice<br/>Mice, Transgenic<br/>Muscular Dystrophy, Oculopharyngeal--genetics<br/>Poly(A)-Binding Protein I--genetics<br/>Trinucleotide Repeat Expansion