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13 in total

1. Inhibition of endoplasmic reticulum-associated degradation rescues native folding in loss of function protein misfolding diseases.

Authors: Fan Wang; Wensi Song; Giovanna Brancati; Laura Segatori
Journal: J Biol Chem Date: 2011-10-17 Impact factor: 5.157

2. Enhancement of drug delivery: enzyme-replacement therapy for murine Morquio A syndrome.

Authors: Shunji Tomatsu; Adriana M Montaño; Vu Chi Dung; Amiko Ohashi; Hirotaka Oikawa; Toshihiro Oguma; Tadao Orii; Luis Barrera; William S Sly
Journal: Mol Ther Date: 2010-03-23 Impact factor: 11.454

Review 3. Emptying the stores: lysosomal diseases and therapeutic strategies.

Authors: Frances M Platt
Journal: Nat Rev Drug Discov Date: 2017-11-17 Impact factor: 84.694

Review 4. Identification and characterization of pharmacological chaperones to correct enzyme deficiencies in lysosomal storage disorders.

Authors: Kenneth J Valenzano; Richie Khanna; Allan C Powe; Robert Boyd; Gary Lee; John J Flanagan; Elfrida R Benjamin
Journal: Assay Drug Dev Technol Date: 2011-06 Impact factor: 1.738

5. Systemic administration of AAV8-α-galactosidase A induces humoral tolerance in nonhuman primates despite low hepatic expression.

Authors: Jennifer B Nietupski; Gregory D Hurlbut; Robin J Ziegler; Qiuming Chu; Bradley L Hodges; Karen M Ashe; Mark Bree; Seng H Cheng; Richard J Gregory; John Marshall; Ronald K Scheule
Journal: Mol Ther Date: 2011-06-28 Impact factor: 11.454

6. Digital microfluidic platform for multiplexing enzyme assays: implications for lysosomal storage disease screening in newborns.

Authors: Ramakrishna S Sista; Allen E Eckhardt; Tong Wang; Carrie Graham; Jeremy L Rouse; Scott M Norton; Vijay Srinivasan; Michael G Pollack; Adviye A Tolun; Deeksha Bali; David S Millington; Vamsee K Pamula
Journal: Clin Chem Date: 2011-08-22 Impact factor: 8.327

7. Multiplex newborn screening for Pompe, Fabry, Hunter, Gaucher, and Hurler diseases using a digital microfluidic platform.

Authors: Ramakrishna S Sista; Tong Wang; Ning Wu; Carrie Graham; Allen Eckhardt; Theodore Winger; Vijay Srinivasan; Deeksha Bali; David S Millington; Vamsee K Pamula
Journal: Clin Chim Acta Date: 2013-05-07 Impact factor: 3.786

8. Rapid assays for Gaucher and Hurler diseases in dried blood spots using digital microfluidics.

Authors: Ramakrishna S Sista; Tong Wang; Ning Wu; Carrie Graham; Allen Eckhardt; Deeksha Bali; David S Millington; Vamsee K Pamula
Journal: Mol Genet Metab Date: 2013-03-24 Impact factor: 4.797

9. Current and emerging treatments and surgical interventions for Morquio A syndrome: a review.

Authors: Shunji Tomatsu; William G Mackenzie; Mary C Theroux; Robert W Mason; Mihir M Thacker; Thomas H Shaffer; Adriana M Montaño; Daniel Rowan; William Sly; Carlos J Alméciga-Díaz; Luis A Barrera; Yasutsugu Chinen; Eriko Yasuda; Kristen Ruhnke; Yasuyuki Suzuki; Tadao Orii
Journal: Res Rep Endocr Disord Date: 2012-12

Review 10. Pharmacological Chaperone Therapy: Preclinical Development, Clinical Translation, and Prospects for the Treatment of Lysosomal Storage Disorders.

Authors: Giancarlo Parenti; Generoso Andria; Kenneth J Valenzano
Journal: Mol Ther Date: 2015-04-16 Impact factor: 11.454