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Do Hung · SciRah

Researcher profile · Semantic Scholar

Do Hung

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Do Hung — researcher profile on SciRah

h-index: 11
Works: 44
Citations: 670

Most-cited papers

The iminosugar isofagomine increases the activity of N370S mutant acid β-glucosidase in Gaucher fibroblasts by several mechanisms
R. Steet, S. Chung, B. Wustman, A. Powe +2 more
2006Proceedings of the National Academy of Sciences of the United States of America219 citations Open access
Semantic Scholar
The pharmacological chaperone 1‐deoxynojirimycin increases the activity and lysosomal trafficking of multiple mutant forms of acid alpha‐glucosidase
J. Flanagan, Barbara Rossi, K. Tang, Xiao-Yang Wu +12 more
2009Human Mutation97 citations Open access
Semantic Scholar
Improved efficacy of a next-generation ERT in murine Pompe disease.
Su Xu, Lun Yi, M. Frascella, Anadina Garcia +15 more
2019JCI Insight79 citations Open access
Semantic Scholar
Challenges in treating Pompe disease: an industry perspective.
Do Hung, Richie Khanna, R. Gotschall
2019Annals of Translational Medicine68 citations Open access
Semantic Scholar
Human Mannose 6-Phosphate-uncovering Enzyme Is Synthesized as a Proenzyme That Is Activated by the Endoprotease Furin*
Do Hung, Wang-Sik Lee, P. Ghosh, Tracy Hollowell +2 more
2002Journal of Biological Chemistry48 citations Open access
Semantic Scholar
Selective action of the iminosugar isofagomine, a pharmacological chaperone for mutant forms of acid-beta-glucosidase.
R. Steet, S. Chung, Wang-Sik Lee, C. Pine +2 more
2007Biochemical Pharmacology45 citations Open access
Semantic Scholar
The Pharmacological Chaperone AT2220 Increases the Specific Activity and Lysosomal Delivery of Mutant Acid Alpha-Glucosidase, and Promotes Glycogen Reduction in a Transgenic Mouse Model of Pompe Disease
Richie Khanna, A. Powe, Lun Yi, R. Soska +12 more
2014PLoS ONE37 citations Open access
Semantic Scholar
Immune transgene-dependent myocarditis in macaques after systemic administration of adeno-associated virus expressing human acid alpha-glucosidase
J. Hordeaux, A. Ramezani, S. Tuske, Nickita Mehta +11 more
2023Frontiers in Immunology21 citations Open access
Semantic Scholar
Endolysosomal N-glycan processing is critical to attain the most active form of the enzyme acid alpha-glucosidase
N. Selvan, Nickita Mehta, S. Venkateswaran, N. Brignol +12 more
2021Journal of Biological Chemistry21 citations Open access
Semantic Scholar
Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease
Barry J. Byrne, G. Parenti, B. Schoser, A. T. van der Ploeg +13 more
2024Frontiers in Neurology16 citations Open access
Semantic Scholar
Failure of Autophagy in Pompe Disease
Do Hung, Naresh K. Meena, Nina Raben
2024Biomolecules11 citations Open access
Semantic Scholar
Plasma total GAA protein PK profiles differ between cipaglucosidase alfa/miglustat and alglucosidase alfa
F. Johnson, Jia Kang, J. Mondick, S. Sitaraman +4 more
2022Molecular Genetics and Metabolism5 citations
Semantic Scholar
Novel recombinant human acid α-glucosidase with optimal glycosylation is significantly better than standard of care enzyme replacement for glycogen clearance in skeletal muscles of GAA knock-out mice
R. Gotschall, Su Xu, Lun Yi, A. Schilling +9 more
20155 citations
Semantic Scholar
114. Pharmacological chaperone therapy for Gaucher disease: Mechanism of action, a survey of responsive mutations and phase I clinical trial results
B. Wustman, C. Pine, Brian Ranes, J. Flanagan +9 more
20084 citations
Semantic Scholar
M021: A novel drug candidate for Pompe disease
R. Gotschall, Kylie Gray, Michael DiGruccio, Jonathan Roberts +6 more
2025Molecular Genetics and Metabolism1 citations
Semantic Scholar
Novel dual promoter AAV gene therapy platform ensures production of therapeutic soluble lysosomal enzymes with high M6P content to enable broad cellular uptake and cross correction
Andrew C. Hedman, Jennifer Srnak, Yi-Cheng Zhao, Shou-nian Liu +3 more
2023Molecular Genetics and Metabolism1 citations
Semantic Scholar
Development of a novel gene therapy for Fabry disease: Engineered alpha-galactosidase A transgene for improved stability
T. Willer, J. Hordeaux, P. Tsai, Daniel Ellsworth +11 more
20211 citations
Semantic Scholar
METABOLIC MYOPATHIES II
Lun Yi, Su Xu, R. Soska, Anju M. Nair +9 more
2018Neuromuscular Disorders1 citations
Semantic Scholar
Histological examination of the effect of a highly phosphorylated proprietary recombinant human acid alpha-glucosidase on glycogen reduction in disease-relevant muscles of Pompe mice
Lun Yi, Su Xu, R. Gotschall, A. Schilling +8 more
20151 citations
Semantic Scholar
GAA engineered to improve cellular uptake enhances correction in a preclinical hematopoietic stem cell gene therapy model of Pompe disease
S. Wantuch, Steven J. Tuske, Sara Benedetti, Nickita Mehta +12 more
2026Molecular therapy. Advances0 citations Open access
Semantic Scholar
M021: Unlocking the future of Pompe ERT
R. Gotschall, Kylie Gray, Naresh K. Meena, Michael DiGruccio +3 more
2026Molecular Genetics and Metabolism0 citations Open access
Semantic Scholar
A novel technology-driven enzyme replacement therapy for Pompe disease
Naresh K. Meena, Kylie Gray, Jonathan Roberts, Michael DiGruccio +5 more
2026Molecular Therapy Advances0 citations Open access
Semantic Scholar
What is in store for Pompe disease therapy?
Do Hung, Kylie Gray, Naresh K. Meena, Michael DiGruccio +6 more
2025Molecular Genetics and Metabolism0 citations
Semantic Scholar
Corrigendum: Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease
Barry J. Byrne, G. Parenti, B. Schoser, A. T. van der Ploeg +13 more
2025Frontiers in Neurology0 citations Open access
Semantic Scholar
Optimized dual promoter AAV gene therapy for lysosomal β-glucocerebrosidase with high M6P content for treatment of neuronopathic Gaucher disease
Andrew Hedman, Steven B. Ortmeier, Shou-nian Liu, Jennifer Srnak +5 more
2024Molecular Genetics and Metabolism0 citations
Semantic Scholar
Hyperactive GlcNAc-1-Phosphotransferase (S1S3 PTase) dramatically increases M6P levels on lysosomal enzymes for substantially improved receptor binding and cellular uptake
M. Digruccio, Kylie Gray, Riley Marcinczyk, Jonathan Roberts +10 more
2024Molecular Genetics and Metabolism0 citations Open access
Semantic Scholar
Targeted protein degradation in lysosome utilizing naturally produced bifunctional antibodies with high levels of mannose 6-phosphate glycans
Andrew Hedman, Shou-nian Liu, Jennifer Srnak, Riley Marcinczyk +5 more
2024bioRxiv0 citations Open access
Semantic Scholar
Evaluation of the effects of HexM and its mannose-6-hyper-phosphorylated form (PhosHexM) in reducing GM2 ganglioside storage in a Tay-Sachs disease mouse model
Mehrafarin Ashiri, Emily N Barker, Wenxuan Wang, Do Hung +6 more
2024Molecular Genetics and Metabolism0 citations
Semantic Scholar
M021 (rhGAA) has a unique glycosylation profile which enables more efficient glycogen reduction and may allow for alternative Pompe ERT dosing strategies
Kylie Gray, Michael DiGruccio, Jonathan Roberts, Vaughn Weaver +11 more
2024Molecular Genetics and Metabolism0 citations
Semantic Scholar
An innovative gene therapy approach to produce novel GALC variant with enhanced protein stability and enzyme activity with high levels of mannose 6-phosphate for Krabbe disease
Do Hung, Mark Sands, Yi-Cheng Zhao, Andrew Hedman +3 more
2024Molecular Genetics and Metabolism0 citations
Semantic Scholar
Highly phosphorylated β-glucocerebrosidase (M011) has much broader tissue targeting and superior substrate reduction with potential for alternative dosing strategies for the treatment of Gaucher disease type I
Do Hung, Kylie Gray, M. Digruccio, Riley Marcinczyk +4 more
2023Molecular Genetics and Metabolism0 citations
Semantic Scholar
M021: rhGAA with optimal glycosylation profile containing very high levels of bis-phosphorylated N-glycans clears accumulated glycogen and rapidly normalizes muscle strength in treated Pompe disease mice
R. Gotschall, M. Digruccio, Kylie Gray, Linda Lyons +12 more
2023Molecular Genetics and Metabolism0 citations
Semantic Scholar
Highly phosphorylated β-glucocerebrosidase (M011) that targets central nervous system neurons as a potential treatment for neuronopathic Gaucher disease type 2 and 3
M. Digruccio, Steven B. Ortmeier, Udayanga S Wanninayake, Jonathan D. Roberts +3 more
2023Molecular Genetics and Metabolism0 citations
Semantic Scholar
Novel AAV gene therapy produces beta-glucocerebrosidase with high levels of M6P to enable cellular uptake and cross-correction in the CNS as a potential treatment for type 2/3 Gaucher disease
Lin Liu, Yi-Cheng Zhao, Andrew C. Hedman, Jennifer Srnak +3 more
2023Molecular Genetics and Metabolism0 citations
Semantic Scholar
M011: A novel highly phosphorylated β-glucocerebrosidase enzyme with broader tissue biodistribution for the treatment of Gaucher disease
R. Gotschall, Lin Liu, M. Digruccio, Udayanga S Wanninayake +7 more
2022Molecular Genetics and Metabolism0 citations
Semantic Scholar
M021: A uniquely glycosylated, highly phosphorylated acid-alpha glucosidase enzyme replacement therapy for the treatment of Pompe disease
R. Gotschall, Lin Liu, Kylie Gray, Nick Wessler +7 more
2022Molecular Genetics and Metabolism0 citations
Semantic Scholar
A major advance in the search for more effective therapy for Pompe disease
N. Raben, E. Ralston, Lun Yi, A. Ponery +7 more
20180 citations
Semantic Scholar
A next-generation Fabry enzyme replacement therapy: A proprietary human α-galactosidase A co-formulated with a pharmacological chaperone, AT1001, shows greater substrate reduction than standard of care in Fabry mice
Su Xu, A. Schilling, Nestor Gomez, M. Frascella +12 more
20180 citations
Semantic Scholar
A novel recombinant human acid alpha-glucosidase, ATB200, leads to greater substrate reduction and improvement in Pompe disease-relevant markers compared to alglucosidase alfa in GAA KO mice
Lun Yi, Su Xu, R. Soska, Anju M. Nair +6 more
20170 citations
Semantic Scholar
Stabilized next-generation recombinant human acid alpha-glucosidase ATB200 clears accumulated glycogen and reverses cellular dysfunction to increase functional muscle strength in a mouse model of Pompe disease
Do Hung, Lun Yi, Su Xu, R. Soska +9 more
20170 citations
Semantic Scholar
Co-administration of the pharmacological chaperone AT2221 with a proprietary recombinant human acid alfa-glucosidase leads to greater plasma exposure and substrate reduction compared to alglucosidase alfa
Richie Khanna, Su Xu, D. Hilliard, Lun Yi +13 more
20160 citations
Semantic Scholar
Chemical conjugation of targeting peptide to ERT substantially improves binding for intended receptor and in vivo efficacy for clearing accumulated substrate in mouse models
Do Hung, R. Gotschall
20140 citations Open access
Semantic Scholar
Beta-glucocerebrosidase recombinant proteins, variants with increased stability and increased catalytic activity retained
Do Hung
20110 citations
Semantic Scholar
23. Pharmacological chaperone treatment for Pompe disease
Do Hung, J. Flanagan, Xiao-Yang Wu, A. Powe +9 more
20080 citations
Semantic Scholar

Frequent co-authors

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R. Gotschall×22
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R. Gotschall — researcher profile on SciRah
K. Valenzano×12
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K. Valenzano — researcher profile on SciRah
Richie Khanna×11
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Richie Khanna — researcher profile on SciRah
Su Xu×10
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Su Xu — researcher profile on SciRah

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M. Frascella — researcher profile on SciRah

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R. Soska — researcher profile on SciRah

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Kylie Gray — researcher profile on SciRah

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Lin Liu — researcher profile on SciRah

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Lun Yi — researcher profile on SciRah

Anadina Garcia×9

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Anadina Garcia — researcher profile on SciRah

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Jessie Feng — researcher profile on SciRah

Riley Marcinczyk×8

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Riley Marcinczyk — researcher profile on SciRah