Keeler, Allison M <br/><br/>
Systemic Delivery of AAVB1-GAA Clears Glycogen and Prolongs Survival in a Mouse Model of Pompe Disease.  [electronic resource] 

 -  Human gene therapy  01 2019 
 - 57-68 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>1557-7422 
<br/><br/><label>Standard No.: </label>10.1089/hum.2018.016  doi 
<br/><br/><label>Subjects--Topical Terms: </label><br/>Animals<br/>Dependovirus--genetics<br/>Disease Models, Animal<br/>Enzyme Activation<br/>Gene Expression<br/>Gene Transfer Techniques<br/>Genetic Therapy--methods<br/>Genetic Vectors--administration & dosage<br/>Glycogen--metabolism<br/>Glycogen Storage Disease Type II--genetics<br/>Humans<br/>Immunohistochemistry<br/>Mice<br/>Mice, Transgenic<br/>Muscle, Skeletal--metabolism<br/>Prognosis<br/>Treatment Outcome<br/>alpha-Glucosidases--genetics