Lenders, Malte <br/><br/>
Mutation-specific Fabry disease patient-derived cell model to evaluate the amenability to chaperone therapy.  [electronic resource] 

 -  Journal of medical genetics  08 2019 
 - 548-556 p.  digital 
<br/><br/> Publication Type: Journal Article; Research Support, Non-U.S. Gov't 
<br/><br/><label>ISSN: </label>1468-6244 
<br/><br/><label>Standard No.: </label>10.1136/jmedgenet-2019-106005  doi 
<br/><br/><label>Subjects--Topical Terms: </label><br/>1-Deoxynojirimycin--administration & dosage<br/>Cell- and Tissue-Based Therapy--methods<br/>Enzyme Replacement Therapy--methods<br/>Fabry Disease--genetics<br/>Gene Editing<br/>HEK293 Cells<br/>Humans<br/>Molecular Chaperones--administration & dosage<br/>Precision Medicine--methods<br/>Trihexosylceramides--metabolism<br/>alpha-Galactosidase--genetics