1. Pharmacological Chaperones: Design and Development of New Therapeutic Strategies for the Treatment of Conformational Diseases
   Marino Convertino et al, 2016, ACS Chem. Biol. CrossRef
2. Newborn Screening for Krabbe Disease and Other Lysosomal Storage Disorders: Broad Lessons Learned
   Joseph Orsini et al, 2017, IJNS CrossRef
3. Pharmaceutical Chaperones and Proteostasis Regulators in the Therapy of Lysosomal Storage Disorders: Current Perspective and Future Promises
   Fedah E. Mohamed et al, 2017, Front. Pharmacol. CrossRef
4. null
   Mahin Khatami, 2017 CrossRef
5. Autophagy and Alzheimer's Disease: From Molecular Mechanisms to Therapeutic Implications.
   Md Sahab Uddin et al, 2018, Front Aging Neurosci CrossRef
6. Three-layer poly(methyl methacrylate) microsystem for analysis of lysosomal enzymes for diagnostic purposes
   Radoslaw Kwapiszewski et al, 2015, Analytica Chimica Acta CrossRef
7. A new type of pharmacological chaperone for G M1 -gangliosidosis related human lysosomal β-galactosidase: N -Substituted 5-amino-1-hydroxymethyl-cyclopentanetriols
   Michael Schalli et al, 2017, Bioorganic & Medicinal Chemistry Letters CrossRef
8. New Approaches to Tay-Sachs Disease Therapy
   Valeriya V. Solovyeva et al, 2018, Front. Physiol. CrossRef
9. Enzyme enhancement therapeutics for lysosomal storage diseases: Current status and perspective
   Ryan Thomas et al, 2018, Molecular Genetics and Metabolism CrossRef
10. null
    Z. Begum Yagci et al, 2018 CrossRef
11. null
    José Ángel Fernández-Higuero et al, 2018 CrossRef
12. Cigarette smoke extract may induce lysosomal storage disease-like adverse health effects
    Eun-Jung Park et al, 2018, J Appl Toxicol CrossRef
13. Lysosomal storage disorders affecting the heart: a review
    Vidhya Nair et al, 2018, Cardiovascular Pathology CrossRef
14. The Role of Dimethyl Sulfoxide (DMSO) in Gene Expression Modulation and Glycosaminoglycan Metabolism in Lysosomal Storage Disorders on an Example of Mucopolysaccharidosis
    Marta Moskot et al, 2019, IJMS CrossRef
15. Hematopoietic stem cell transplantation for mucopolysaccharidoses; past, present, and future
    Madeleine Taylor et al, 2019, Biology of Blood and Marrow Transplantation CrossRef
16. The role of autophagy in neurodegenerative disease
    Ralph A Nixon, 2013, Nat Med CrossRef
17. The glycosylation design space for recombinant lysosomal replacement enzymes produced in CHO cells
    Weihua Tian et al, 2019, Nat Commun CrossRef
18. Biologically active branched-chain aminocyclopentane tetraols from d-galactose
    Michael Schalli et al, 2019, Monatsh Chem CrossRef
19. Nanomaterial designing strategies related to cell lysosome and their biomedical applications: A review
    Bhowmira Rathore et al, 2019, Biomaterials CrossRef
20. null
    Maurizio Scarpa et al, 2015 CrossRef
21. Effects of gene therapy on cardiovascular symptoms of lysosomal storage diseases
    Edina Poletto et al, 2019, Genet. Mol. Biol. CrossRef
22. null
    Maurizio Scarpa et al, 2016 CrossRef
23. Scaffolds for Sustained Release of Ambroxol Hydrochloride, a Pharmacological Chaperone That Increases the Activity of Misfolded β-Glucocerebrosidase
    Hamidreza Enshaei et al, 2019, Macromol. Biosci. CrossRef
24. Respuesta
    Carme Costa et al, 2013, Medicina Clínica CrossRef
25. Blood–brain barrier structure and function and the challenges for CNS drug delivery
    N. Joan Abbott, 2013, J Inherit Metab Dis CrossRef
26. C-Branched Iminosugars: α-Glucosidase Inhibition by Enantiomers of isoDMDP, isoDGDP, and isoDAB–l-isoDMDP Compared to Miglitol and Miglustat
    Sarah F. Jenkinson et al, 2013, J. Org. Chem. CrossRef
27. Pharmacotherapy of Pompe disease
    Giancarlo Parenti et al, 2013, Expert Opinion on Orphan Drugs CrossRef
28. Corrective GUSB Transfer to the Canine Mucopolysaccharidosis VII Brain
    Aurelie Cubizolle et al, 2014, Molecular Therapy CrossRef
29. A Systematic Investigation of Iminosugar Click Clusters as Pharmacological Chaperones for the Treatment of Gaucher Disease
    Antoine Joosten et al, 2014, ChemBioChem CrossRef
30. Targeted delivery of pharmacological chaperones for Gaucher disease to macrophages by a mannosylated cyclodextrin carrier
    Julio Rodríguez-Lavado et al, 2014, Org. Biomol. Chem. CrossRef
31. Innovative strategies to treat protein misfolding in inborn errors of metabolism: pharmacological chaperones and proteostasis regulators
    Ania C. Muntau et al, 2014, J Inherit Metab Dis CrossRef
32. Lentiviral vectors containing mouse Csf1r control elements direct macrophage-restricted expression in multiple species of birds and mammals
    Clare Pridans et al, 2014, Molecular Therapy - Methods & Clinical Development CrossRef
33. Pharmacological chaperones increase residual β-galactocerebrosidase activity in fibroblasts from Krabbe patients
    Anna Sara Berardi et al, 2014, Molecular Genetics and Metabolism CrossRef
34. ERdj3 Is an Endoplasmic Reticulum Degradation Factor for Mutant Glucocerebrosidase Variants Linked to Gaucher’s Disease
    Yun Lei Tan et al, 2014, Chemistry & Biology CrossRef
35. Pharmacological chaperone therapy for lysosomal storage diseases
    Giancarlo Parenti et al, 2014, Future Medicinal Chemistry CrossRef
36. Non-neuronopathic lysosomal storage disorders: Disease spectrum and treatments
    Gregory M. Pastores et al, 2015, Best Practice & Research Clinical Endocrinology & Metabolism CrossRef
37. Protein trafficking defects in inherited kidney diseases
    C. Schaeffer et al, 2014, Nephrology Dialysis Transplantation CrossRef
38. Unlocking a Caged Lysosomal Protein from a Polymeric Nanogel with a pH Trigger
    Mijanur Rahaman Molla et al, 2014, Biomacromolecules CrossRef
39. The clinical management of type 2 Gaucher disease
    Karin Weiss et al, 2015, Molecular Genetics and Metabolism CrossRef
40. Neuronopathic lysosomal storage disorders: Approaches to treat the central nervous system
    Maurizio Scarpa et al, 2015, Best Practice & Research Clinical Endocrinology & Metabolism CrossRef
41. Expanded newborn screening by mass spectrometry: New tests, future perspectives
    Daniela Ombrone et al, 2016, Mass Spec Rev CrossRef
42. Prospective therapies for mucopolysaccharidoses
    Anna Tylki-Szymańska et al, 2015, Expert Opinion on Orphan Drugs CrossRef
43. CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease
    John Marshall et al, 2016, Molecular Therapy CrossRef
44. The role of autophagy in neurodegenerative diseases
    I. A. Kochergin et al, 2016, Neurochem. J. CrossRef
45. null
    Arnold E. Stütz et al, 2016 CrossRef
46. Lysosomal Re-acidification Prevents Lysosphingolipid-Induced Lysosomal Impairment and Cellular Toxicity
    Christopher J. Folts et al, 2016, PLoS Biol CrossRef
47. Aspartylglycosaminuria: a review
    Maria Arvio et al, 2016, Orphanet J Rare Dis CrossRef
48. Temporary Efficacy of Pyrimethamine in Juvenile-Onset Tay-Sachs Disease Caused by 2 Unreported HEXA Mutations in the Indian Population
    Anaita Udwadia-Hegde et al, 2017, Child Neurology Open CrossRef
49. N-Substituted 5-amino-1-hydroxymethyl-cyclopentanetriols: A new family of activity promotors for a G M1 -gangliosidosis related human lysosomal β-galactosidase mutant
    Michael Schalli et al, 2017, Carbohydrate Research CrossRef
50. Long-term enzyme replacement therapy improves neurocognitive functioning and hippocampal synaptic plasticity in immune-tolerant alpha-mannosidosis mice
    Stijn Stroobants et al, 2017, Neurobiology of Disease CrossRef
51. null
    Subrata Ganguli et al, 2017 CrossRef
52. Targeted delivery of lysosomal enzymes to the endocytic compartment in human cells using engineered extracellular vesicles
    Mai Anh Do et al, 2019, Sci Rep CrossRef
53. Lysosomal size matters
    Mariana E. G. Araujo et al, 2019, Traffic CrossRef
54. null
    Shyamasree Ghosh, 2020 CrossRef
55. Pre-clinical Mouse Models of Neurodegenerative Lysosomal Storage Diseases
    Jacob M. Favret et al, 2020, Front. Mol. Biosci. CrossRef
56. Highlights on Genomics Applications for Lysosomal Storage Diseases
    Valentina La Cognata et al, 2020, Cells CrossRef
57. Mechanistic Insights into the Chaperoning of Human Lysosomal-Galactosidase Activity: Highly Functionalized Aminocyclopentanes and C-5a-Substituted Derivatives of 4-epi-Isofagomine
    Patrick Weber et al, 2020, Molecules CrossRef
58. Deregulation of signalling in genetic conditions affecting the lysosomal metabolism of cholesterol and galactosyl-sphingolipids
    S. Gowrishankar et al, 2020, Neurobiology of Disease CrossRef
59. Cerliponase alfa for CLN2 disease, a promising therapy
    Shawn C. Aylward et al, 2020, Expert Opinion on Orphan Drugs CrossRef
60. New treatment for myocardial diseases
    Kenji Onoue et al, 2020, Journal of Cardiology CrossRef
61. Aspartylglucosaminuria: Clinical Presentation and Potential Therapies
    Kimberly Goodspeed et al, 2021, J Child Neurol CrossRef
62. Potential Treatment of Lysosomal Storage Disease through Modulation of the Mitochondrial—Lysosomal Axis
    Myeong Uk Kuk et al, 2021, Cells CrossRef
63. Enzyme replacement combinational therapy: Effective treatments for mucopolysaccharidoses
    Azam Safary et al, 2021, Expert Opinion on Biological Therapy CrossRef
64. Chemoenzymatic glycan-selective remodeling of a therapeutic lysosomal enzyme with high-affinity M6P-glycan ligands. Enzyme substrate specificity is the name of the game
    Xiao Zhang et al, 2021, Chem. Sci. CrossRef
65. Compromised Catalysis and Potential Folding Defects in in Vitro Studies of Missense Mutants Associated with Hereditary Phosphoglucomutase 1 Deficiency
    Yingying Lee et al, 2014, Journal of Biological Chemistry CrossRef
66. Tween® Preserves Enzyme Activity and Stability in PLGA Nanoparticles
    Jason Thomas Duskey et al, 2021, Nanomaterials CrossRef
67. Reproduction in Animal Models of Lysosomal Storage Diseases: A Scoping Review
    Daniela Vuolo et al, 2021, Front. Mol. Biosci. CrossRef
68. Pharmacological Chaperones for β‐Galactosidase Related to G M1 ‐Gangliosidosis and Morquio B: Recent Advances
    Arnold E. Stütz et al, 2021, Chem. Rec. CrossRef
69. Therapeutic Approaches in Lysosomal Storage Diseases
    Carlos Fernández-Pereira et al, 2021, Biomolecules CrossRef
70. Molecular environment and atypical function: What do we know about enzymes associated with Mucopolysaccharidoses?
    Weijing Kong et al, 2022, Orphanet J Rare Dis CrossRef
71. GCase Enhancers: A Potential Therapeutic Option for Gaucher Disease and Other Neurological Disorders
    Macarena Martínez-Bailén et al, 2022, Pharmaceuticals CrossRef
72. Current treatment options and novel nanotechnology-driven enzyme replacement strategies for lysosomal storage disorders
    Ambra Del Grosso et al, 2022, Advanced Drug Delivery Reviews CrossRef
73. Rare lysosomal disease registries: lessons learned over three decades of real-world evidence
    P. K. Mistry et al, 2022, Orphanet J Rare Dis CrossRef
74. null
    , 2021 CrossRef
75. Abnormal Pre-mRNA Splicing in Exonic Fabry Disease-Causing GLA Mutations
    Franziska Alfen et al, 2022, IJMS CrossRef
76. Development of Lanzyme as the Potential Enzyme Replacement Therapy Drug for Fabry Disease
    Mulan Deng et al, 2022, Biomolecules CrossRef
77. The Regulatory Role and Re-search Progress of TFEB in Lysosomal Storage Disease
    士雪 程, 2023, BP CrossRef
78. Positive Impact of Home ERT for Mucopolysaccharidoses and Pompe Disease: The Lesson Learnt from the COVID-19 Pandemic
    Agata Fiumara et al, 2023, Healthcare CrossRef
79. CRISPR Technologies in Chinese Hamster Ovary Cell Line Engineering
    Katja Glinšek et al, 2023, IJMS CrossRef
80. Unlocking cellular barriers: silica nanoparticles and fullerenol conjugated cell-penetrating agents for enhanced intracellular drug delivery
    Eduardo Ravelo-Nieto et al, 2023, Front. Bioeng. Biotechnol. CrossRef
81. Glycosylation shapes the efficacy and safety of diverse protein, gene and cell therapies
    Frances Rocamora et al, 2023, Biotechnology Advances CrossRef
82. null
    Esteban Alberto Gonzalez et al, 2023 CrossRef
83. Functional Characterization of a Spectrum of Genetic Variants in a Family with Succinic Semialdehyde Dehydrogenase Deficiency
    Miroslava Didiasova et al, 2024, IJMS CrossRef
84. Evidence of Lysosomal β-Hexosaminidase Enzymatic Activity Associated with Extracellular Vesicles: Potential Applications for the Correction of Sandhoff Disease
    Eleonora Calzoni et al, 2024, JFB CrossRef
85. Lysosomes in the immunometabolic reprogramming of immune cells in atherosclerosis
    Fabrizia Bonacina et al, 2024, Nat Rev Cardiol CrossRef
86. null
    Sushil Sharma, 2025 CrossRef