Please confirm you are human (Sign Up for free to never see this)
← Back to Search
Investigation Of Vesicular-Mediated Transport Of Intercellular Adhesion Molecule-1-Targeted Carriers For Treatment Of Lysosomal Storage Disorders
R. L. Manthe
Published 2017 · Materials Science
Save to my Library
Download PDFAnalyze on Scholarcy
Title of Dissertation: INVESTIGATION OF VESICULARMEDIATED TRANSPORT OF INTERCELLULAR ADHESION MOLECULE1-TARGETED CARRIERS FOR TREATMENT OF LYSOSOMAL STORAGE DISORDERS Rachel Lee Manthe, Doctor of Philosophy, 2017 Dissertation directed by: Associate Professor, Silvia Muro Fischell Department of Bioengineering & Institute for Bioscience and Biotechnology Research Numerous cellular processes and therapeutic interventions rely on vesicularmediated endocytosis to gain entry into cells and sub-cellular compartments, as well as for transcellular transport across biological barriers such as found at the blood-brain interface. Yet, endocytic behavior can be altered in disease, representing an additional hurdle in the design of effective therapeutic strategies. Lysosomal storage disorders (LSDs), characterized by lysosomal accumulation of undigested substrates as a result of deficient enzymatic activity, illustrate this paradigm. Currently, intravenous infusion of recombinant lysosomal enzymes to replace those deficient is the standard clinical approach for these disorders. However, clathrin-mediated endocytosis utilized by replacement enzymes for cellular uptake and lysosomal trafficking is altered, thereby impacting treatment efficacy as recently demonstrated in acid sphingomyelinase-deficient type A Niemann-Pick disease (NPD). Therefore, alternative means to bypass defunct routes is warranted. Therapeutic delivery via polymer nanocarriers targeting intercellular adhesion molecule-1 (anti-ICAM NCs), a cell-surface molecule overexpressed in endothelial and subjacent tissue cells during inflammation, such as in LSDs, represents a viable option since it permits uptake, intraand transcellular transport via a unique endocytic route called the cell adhesion molecule (CAM) pathway. In this dissertation, cell culture and animal models were used to examine the (1) endocytic activity of the CAM pathway and other clathrin-independent routes in type A NPD, (2) role of targeting valency (i.e., density of ICAM-1-targeting molecules on the NC surface) in regulating the CAM pathway, and (3) effects induced via engagement of ICAM-1 on cells by anti-ICAM NCs. The results herein demonstrate the CAM pathway is more active in diseased cells compared to other classical endocytic pathways, making it the most amenable route for therapeutic enzyme replacement. Further, modulating targeting valency of NCs optimized this strategy for enhanced enzyme delivery to the brain, a target organ for type A NPD. Lastly, anti-ICAM NCs attenuated endothelial release of soluble ICAM-1, an inflammatory regulator, representing a secondary benefit of this system. Overall, this work validates utility of anti-ICAM NCs for enzyme replacement to treat NPD and likely other LSDs, and provides insight into biological processes and design parameters that influence the therapeutic efficacy of targeted drug carriers. INVESTIGATION OF VESICULAR-MEDIATED TRANSPORT OF INTERCELLULAR ADHESION MOLECULE-1-TARGETED CARRIERS FOR TREATMENT OF LYSOSOMAL STORAGE DISORDERS
This paper references
The demographics and distribution of type B Niemann-Pick disease: novel mutations lead to new genotype/phenotype correlations.
C. Simonaro (2002)
Lysosomal disorders: from storage to cellular damage.
A. Ballabio (2009)
A fluorescence-based, high-performance liquid chromatographic assay to determine acid sphingomyelinase activity and diagnose types A and B Niemann-Pick disease.
X. He (2003)
The Role of Autophagy in Cancer: Therapeutic Implications
Z. J. Yang (2011)
Hernandez-Trejo, The impact of nanobiotechnology on the development of new drug delivery systems
O. Kayser (2005)
Targeting of the CNS in MPS-IH using a nonviral transferrin-alpha-L-iduronidase fusion gene product.
Mark J. Osborn (2008)
Defining the pathway for Tat-mediated delivery of beta-glucuronidase in cultured cells and MPS VII mice.
K. Orii (2005)
Nanomedicine: current status and future prospects
S. Moghimi (2005)
Identification of seven novel SMPD1 mutations causing Niemann–Pick disease types A and B
P. Irún (2013)
In Search of the Astrocytic Factor(s) Modulating Blood–Brain Barrier Functions in Brain Capillary Endothelial Cells In Vitro
R. Haseloff (2004)
Factors Affecting the Clearance and Biodistribution of Polymeric Nanoparticles
F. Alexis (2008)
Intravascular ultrasound molecular imaging of atheroma components in vivo.
A. Hamilton (2004)
Inhibitors of the V0 subunit of the vacuolar H+-ATPase prevent segregation of lysosomal- and secretory-pathway proteins
J. A. Sobota (2009)
Non-viral systemic delivery of siRNA or antisense oligonucleotides targeted to Jun N-terminal kinase 1 prevents cellular hypoxic damage
S. Betigeri (2010)
Improved intracellular delivery of glucocerebrosidase mediated by the HIV-1 TAT protein transduction domain.
K. Lee (2005)
Advanced drug delivery systems that target the vascular endothelium.
Bi-Sen Ding (2006)
Endothelium in health and disease.
W. Aird (2008)
Enzyme replacement therapy for mucopolysaccharidosis VI: a phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension study.
P. Harmatz (2006)
Nano/micro technologies for delivering macromolecular therapeutics using poly(D,L-lactide-co-glycolide) and its derivatives.
R. C. Mundargi (2008)
Colloidal systems for drug delivery: from design to therapy.
Mariana Beija (2012)
The Pharmacological Chaperone N-butyldeoxynojirimycin Enhances Enzyme Replacement Therapy in Pompe Disease Fibroblasts
Caterina Porto (2009)
Magnetic nanoparticles with dual functional properties: drug delivery and magnetic resonance imaging.
Tapan Jain (2008)
Adhesion receptors of the immune system
T. Springer (1990)
Endocytic mechanisms for targeted drug delivery.
Lisa Bareford (2007)
Polymer therapeutics-prospects for 21st century: the end of the beginning.
R. Duncan (2013)
Endocytosis of nanomedicines.
G. Sahay (2010)
Peptide‐mediated cell penetration and targeted delivery of gold nanoparticles into lysosomes
Chaitali D Dekiwadia (2012)
Biological evaluation of calcium alginate microspheres as a vehicle for the localized delivery of a therapeutic enzyme.
C. Barrias (2005)
Andjelkovic, Brain endothelial cell-cell junctions: how to "open" the blood brain barrier
S. M. Stamatovic (2008)
Lysosomal storage disorders: The cellular impact of lysosomal dysfunction
F. Platt (2012)
Diffusion of univalent ions across the lamellae of swollen phospholipids.
A. Bangham (1965)
Distinct endocytotic pathways in epidermal growth factor-stimulated human carcinoma A431 cells [published erratum appears in J Cell Biol 1990 Mar;110(3):859]
M. A. West (1989)
Enhanced delivery of α-glucosidase for Pompe disease by ICAM-1-targeted nanocarriers: comparative performance of a strategy for three distinct lysosomal storage disorders.
Janet Hsu (2012)
Pro-MMP-9 activation by the MT1-MMP/MMP-2 axis and MMP-3: role of TIMP-2 and plasma membranes.
M. Tóth (2003)
Flow dynamics, binding and detachment of spherical carriers targeted to ICAM-1 on endothelial cells.
A. J. Calderon (2009)
Proteolytic cleavage of ICAM-1 by human neutrophil elastase.
B. Champagne (1998)
Remodeling of cellular cytoskeleton by the acid sphingomyelinase/ceramide pathway
Y. Zeidan (2008)
Slow intracellular trafficking of catalase nanoparticles targeted to ICAM-1 protects endothelial cells from oxidative stress.
S. Muro (2003)
Materials for advanced drug delivery in the 21st century: a focus area for Advanced Drug Delivery Reviews.
H. Ghandehari (2008)
Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase).
J. Wraith (2004)
Nanoparticle transport across the blood brain barrier
A. Grabrucker (2016)
Strategies for delivery of therapeutics into the central nervous system for treatment of lysosomal storage disorders
S. Muro (2012)
Endosomal escape pathways for delivery of biologicals.
Amir K. Varkouhi (2011)
Glycosylation-independent targeting enhances enzyme delivery to lysosomes and decreases storage in mucopolysaccharidosis type VII mice.
J. LeBowitz (2004)
Distinct Subcellular Trafficking Resulting from Monomeric vs Multimeric Targeting to Endothelial ICAM-1: Implications for Drug Delivery
Rasa Ghaffarian (2014)
Autophagy failure in Alzheimer's disease—locating the primary defect
R. Nixon (2011)
Ceramide in bacterial infections and cystic fibrosis
H. Grassmé (2008)
Live dynamic imaging of caveolae pumping targeted antibody rapidly and specifically across endothelium in the lung
P. Oh (2007)
Molecular mechanism and physiological functions of clathrin-mediated endocytosis
H. McMahon (2011)
Guanidinylated neomycin mediates heparan sulfate-dependent transport of active enzymes to lysosomes.
Stéphane Sarrazin (2010)
V-ATPase engagement in autophagic processes
D. Mijaljica (2011)
Lysosomal exocytosis and lipid storage disorders
M. Samie (2014)
Endocytosis and intracellular trafficking as gateways for nanomedicine delivery: opportunities and challenges.
R. Duncan (2012)
Purified intercellular adhesion molecule-1 (ICAM-1) is a ligand for lymphocyte function-associated antigen 1 (LFA-1)
S. D. Marlin (1987)
A Fibrinogen-Derived Peptide Provides Intercellular Adhesion Molecule-1-Specific Targeting and Intraendothelial Transport of Polymer Nanocarriers in Human Cell Cultures and Mice
C. Garnacho (2012)
Endocytosis pathways in endothelium: how many?
R. Stan (2006)
Tumor Necrosis Factor-α-converting Enzyme (TACE/ADAM-17) Mediates the Ectodomain Cleavage of Intercellular Adhesion Molecule-1 (ICAM-1)*
N. Tsakadze (2006)
New biotechnological and nanomedicine strategies for treatment of lysosomal storage disorders.
S. Muro (2010)
Receptor-Mediated Endocytosis and the Sorting of Internalized Proteins
H. Lodish (2000)
864 Polarized secretion of chemokine RANTES and sICAM-1 from a bronchial epithelial cell line BEAS-2B
S. Kinjyo (2000)
Magnetic nanoparticles for theragnostics.
V. Shubayev (2009)
Cyclodextrins in the treatment of a mouse model of Niemann-Pick C disease.
F. Camargo (2001)
Molecular and cellular mechanisms of receptor-mediated endocytosis.
V. Brown (1991)
Matrix metalloproteinases as modulators of inflammation.
A. M. Manicone (2008)
ICAM-1 signaling in endothelial cells.
C. Lawson (2009)
Internalization of cholera toxin by different endocytic mechanisms.
M. Torgersen (2001)
Effects of Polyethylene Glycol Spacer Length and Ligand Density on Folate Receptor Targeting of Liposomal Doxorubicin In Vitro
K. Kawano (2011)
Stimulation of tumor growth by human soluble intercellular adhesion molecule-1.
Y. S. Gho (2001)
Non-inhibitory antibodies impede lysosomal storage reduction during enzyme replacement therapy of a lysosomal storage disease
U. Matzner (2008)
Delivery of Acid Sphingomyelinase in Normal and Niemann-Pick Disease Mice Using Intercellular Adhesion Molecule-1-Targeted Polymer Nanocarriers
C. Garnacho (2008)
Quantum dots as a platform for nanoparticle drug delivery vehicle design.
C. Probst (2013)
Successful within-patient dose escalation of olipudase alfa in acid sphingomyelinase deficiency.
M. Wasserstein (2015)
Differential Effects of Ceramide and Sphingosine 1-Phosphate on ERM Phosphorylation
D. Canals (2010)
Parkinson's Disease and Autophagy
A. Sánchez-Pérez (2012)
Aminabhavi, Recent advances on chitosan-based micro- and nanoparticles in drug delivery
S. A. Agnihotri (2004)
Arylsulfatase A Overexpressing Human iPSC-derived Neural Cells Reduce CNS Sulfatide Storage in a Mouse Model of Metachromatic Leukodystrophy.
J. Doerr (2015)
Biosynthesis of lysosomal hydrolases: their synthesis in bound polysomes and the role of co- and post-translational processing in determining their subcellular distribution
MG Rosenfeld (1982)
Clathrin- and caveolin-1–independent endocytosis
Eva-Maria Damm (2005)
Ethanol enhancement of blood-brain barrier permeability to catecholamines in chicks.
J. Hanig (1972)
Filipin-dependent Inhibition of Cholera Toxin: Evidence for Toxin Internalization and Activation through Caveolae-like Domains
P. Orlandi (1998)
Gene therapy for lysosomal storage diseases: progress, challenges and future prospects.
S. Seregin (2011)
Nanocarriers’ entry into the cell: relevance to drug delivery
H. Hillaireau (2009)
α-Galactosidase-A Loaded-Nanoliposomes with Enhanced Enzymatic Activity and Intracellular Penetration.
I. Cabrera (2016)
Immune response to enzyme replacement therapies in lysosomal storage diseases and the role of immune tolerance induction.
P. Kishnani (2016)
Transcytosis and brain uptake of transferrin-containing nanoparticles by tuning avidity to transferrin receptor
Devin T. Wiley (2013)
Gaucher disease-associated glucocerebrosidases show mutation-dependent chemical chaperoning profiles.
A. Sawkar (2005)
Differing regulation and function of ICAM-1 and class II antigens on renal tubular cells.
A. Jevnikar (1990)
Caveosomes and endocytosis of lipid rafts
B. Nichols (2003)
A block of autophagy in lysosomal storage disorders.
C. Settembre (2008)
Delivery of DNA into muscle for treating systemic diseases: advantages and challenges.
C. Trollet (2008)
At the acidic edge: emerging functions for lysosomal membrane proteins.
E. Eskelinen (2003)
The lysosome and neurodegenerative diseases.
L. Zhang (2009)
A distinct mRNA encoding a soluble form of ICAM-1 molecule expressed in human tissues.
T. Wakatsuki (1995)
CARGO RECOGNITION FAILURE IS RESPONSIBLE FOR INEFFICIENT AUTOPHAGY IN HUNTINGTON’S DISEASE
M. Martinez-Vicente (2010)
Bayever, The status of hematopoietic stem cell transplantation in lysosomal storage disease
J. J. Malatack (2003)
δ-Tocopherol Reduces Lipid Accumulation in Niemann-Pick Type C1 and Wolman Cholesterol Storage Disorders*
Miao Xu (2012)
Endothelial cell–cell junctions: happy together
E. Dejana (2004)
The cell biology of lysosomal storage disorders
A. Futerman (2004)
Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results of a phase I/II trial.
Effects of intracerebroventricular administration of 2-hydroxypropyl-β-cyclodextrin in a patient with Niemann–Pick Type C disease
M. Matsuo (2014)
Trypanosoma cruzi subverts the sphingomyelinase-mediated plasma membrane repair pathway for cell invasion
M. C. Fernandes (2011)
Feasibility and safety of systemic rAAV9-hNAGLU delivery for treating mucopolysaccharidosis IIIB: toxicology, biodistribution, and immunological assessments in primates.
Darren A Murrey (2014)
Enzyme-Replacement Therapies for Lysosomal Storage Diseases
T. Ratko (2013)
Cellular targets and mechanisms in the cytotoxic action of non-biodegradable engineered nanoparticles.
E. Fröhlich (2013)
Steps in the phosphorylation of the high mannose oligosaccharides of lysosomal enzymes.
S. Kornfeld (1982)
Clathrin-independent pinocytosis is induced in cells overexpressing a temperature-sensitive mutant of dynamin
H. Damke (1995)
A human intercellular adhesion molecule (ICAM-1) distinct from LFA-1.
R. Rothlein (1986)
Intercellular adhesion molecules and vascular cell adhesion molecule-1 and the kidney.
R. P. Wuthrich (1992)
Chemical chaperone therapy for GM1-gangliosidosis
Y. Suzuki (2008)
Enhancing biodistribution of therapeutic enzymes in vivo by modulating surface coating and concentration of ICAM-1-targeted nanocarriers.
Janet Hsu (2014)
Mutations in palmitoyl-protein thioesterase 1 alter exocytosis and endocytosis at synapses in Drosophila larvae
E. Aby (2013)
Characterization of endothelial internalization and targeting of antibody-enzyme conjugates in cell cultures and in laboratory animals.
S. Muro (2004)
Protein transduction of Rab9 in Niemann‐Pick C cells reduces cholesterol storage
K. Narita (2005)
[Niemann-Pick disease types A and B].
K. Ohno (1995)
Drug delivery and targeting.
R. Langer (1998)
Shedding of ICAM-1 from human melanoma cell lines induced by IFN-gamma and tumor necrosis factor-alpha. Functional consequences on cell-mediated cytotoxicity.
J. Becker (1991)
Characterization of common SMPD1 mutations causing types A and B Niemann-Pick disease and generation of mutation-specific mouse models.
Iwan G. Jones (2008)
Bonifacino, Sorting of lysosomal proteins
T. Braulke (2009)
Transcriptional Activation of Lysosomal Exocytosis Promotes Cellular Clearance
D. Medina (2011)
Self-assembled nanoplatform for targeted delivery of chemotherapy agents via affinity-regulated molecular interactions.
Spencer Park (2010)
Caveolar endocytosis of simian virus 40 reveals a new two-step vesicular-transport pathway to the ER
L. Pelkmans (2001)
Challenges in polymer therapeutics: state of the art and prospects of polymer drugs.
A. Kabanov (2003)
Tumor vascular permeability and the EPR effect in macromolecular therapeutics: a review.
H. Maeda (2000)
Prevalence of lysosomal storage disorders.
P. Meikle (1999)
Delivery of liposome‐sequestered hydrophobic proteins to lysosomes of normal and batten disease cells
N. Ansari (1997)
Intercellular Adhesion Molecule 1 Engagement Modulates Sphingomyelinase and Ceramide, Supporting Uptake of Drug Carriers by the Vascular Endothelium
Daniel Serrano (2012)
Endothelial Targeting of High-Affinity Multivalent Polymer Nanocarriers Directed to Intercellular Adhesion Molecule 1
S. Muro (2006)
Release of soluble ICAM-1 from human lung fibroblasts, aortic smooth muscle cells, dermal microvascular endothelial cells, bronchial epithelial cells, and keratinocytes.
K. H. Leung (1999)
Macropinocytosis Is the Endocytic Pathway That Mediates Macrophage Foam Cell Formation with Native Low Density Lipoprotein*
H. Kruth (2005)
Interference in Autophagosome Fusion by Rare Earth Nanoparticles Disrupts Autophagic Flux and Regulation of an Interleukin-1β Producing Inflammasome
Ruibin Li (2014)
PLGA nanoparticle--peptide conjugate effectively targets intercellular cell-adhesion molecule-1.
N. Zhang (2008)
Phorbol myristate acetate-mediated stimulation of transcytosis and apical recycling in MDCK cells
M. Cardone (1994)
Vascular Targeting of Nanocarriers: Perplexing Aspects of the Seemingly Straightforward Paradigm
Melissa D. Howard (2014)
Targeted ultrasound contrast agents: in vitro assessment of endothelial dysfunction and multi-targeting to ICAM-1 and sialyl Lewisx.
G. Weller (2005)
Acidic Nanoparticles Are Trafficked to Lysosomes and Restore an Acidic Lysosomal pH and Degradative Function to Compromised ARPE-19 Cells
Gabriel C. Baltazar (2012)
Enzyme replacement and enhancement therapies: lessons from lysosomal disorders
R. Desnick (2003)
Immunoliposomes that target endothelium in vitro are dependent on lipid raft formation.
Rico C Gunawan (2010)
Low density lipoprotein (LDL)-mediated suppression of cholesterol synthesis and LDL uptake is defective in Niemann-Pick type C fibroblasts.
L. Liscum (1987)
Clathrin-independent endocytosis: new insights into caveolae and non-caveolar lipid raft carriers.
M. Kirkham (2005)
Identification of a distinct mutation spectrum in the SMPD1 gene of Chinese patients with acid sphingomyelinase-deficient Niemann-Pick disease
H. Zhang (2013)
Activation of progelatinase B (MMP-9) by gelatinase A (MMP-2).
R. Fridman (1995)
Treatment of lysosomal storage disorders : progress with enzyme replacement therapy.
M. Rohrbach (2007)
Modelling of the blood–brain barrier in drug discovery and development
R. Cecchelli (2007)
Overcoming in vivo barriers to targeted nanodelivery.
A. Chrastina (2011)
Specific Binding, Uptake, and Transport of ICAM-1-Targeted Nanocarriers Across Endothelial and Subendothelial Cell Components of the Blood–Brain Barrier
Janet Hsu (2013)
Selective caveolin-1-dependent endocytosis of glycosphingolipids.
R. D. Singh (2003)
The uptake of enzymes into lysosomes: an overview.
E. Neufeld (1980)
Molecular genetic characterization of novel sphingomyelin phosphodiesterase 1 mutations causing niemann-pick disease.
B. Tóth (2012)
Cyclodextrin Induces Calcium-Dependent Lysosomal Exocytosis
F. Chen (2010)
Historical Fragments; Methods; Some Terminology
E. Holtzman (1989)
Biopharmaceutical drug targeting to the brain
W. Pardridge (2010)
Proteolytic Degradation of VE-Cadherin Alters the Blood-Retinal Barrier in Diabetes
D. Navaratna (2007)
Screening and optimization of ligand conjugates for lysosomal targeting.
I. Meerovich (2011)
Nanoparticles restore lysosomal acidification defects: Implications for Parkinson and other lysosomal-related diseases
M. Bourdenx (2016)
Transendothelial migration of lymphocytes across high endothelial venules into lymph nodes is affected by metalloproteinases.
C. Faveeuw (2001)
Significance of immune response to enzyme-replacement therapy for patients with a lysosomal storage disorder.
D. Brooks (2003)
Bi-directional induction of matrix metalloproteinase-9 and tissue inhibitor of matrix metalloproteinase-1 during T lymphoma/endothelial cell contact: implication of ICAM-1.
F. Aoudjit (1998)
Molecular and Cellular Mechanisms of Ectodomain Shedding
K. Hayashida (2010)
Storage solutions: treating lysosomal disorders of the brain
M. Jeyakumar (2005)
Induced Pluripotent Stem Cells for Disease Modeling and Evaluation of Therapeutics for Niemann-Pick Disease Type A
Y. Long (2016)
Endothelial Cell Adhesion Molecules and Drug Delivery Applications
Helim Aranda-Espinoza (2015)
Uropod elongation is a common final step in leukocyte extravasation through inflamed vessels
Y. Hyun (2012)
Inflammation-Specific T1 Imaging Using Anti-Intercellular Adhesion Molecule 1 Antibody-Conjugated Gadolinium Diethylenetriaminepentaacetic Acid
Kyu-Sil Choi (2007)
Ganglioside GM1-mediated Transcytosis of Cholera Toxin Bypasses the Retrograde Pathway and Depends on the Structure of the Ceramide Domain*
D. Saslowsky (2013)
Dysfunction of endocytic and autophagic pathways in a lysosomal storage disease
T. Fukuda (2006)
Adesnik, The biogenesis of membranes and organelles., in The Metabolic and Molecular Bases of Inherited Disease, C
D. Sabatini (2001)
Low-dose therapy trumps high-dose therapy again in the treatment of Gaucher disease.
A. Zimran (2006)
Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA.
S. Worgall (2008)
Blood-brain barrier delivery.
W. Pardridge (2007)
Endocytosis and the Endolysosomal Route in Drug Delivery
J. Rappaport (2016)
Autophagy and lysosomal dysfunction as emerging mechanisms of nanomaterial toxicity
S. Stern (2012)
Lysosomal storage diseases and the blood-brain barrier.
D. Begley (2008)
Lipoprotein receptor binding, cellular uptake, and lysosomal delivery of fusions between the receptor-associated protein (RAP) and alpha-L-iduronidase or acid alpha-glucosidase.
W. Prince (2004)
Endogenous Ceramide Contributes to the Transcytosis of oxLDL across Endothelial Cells and Promotes Its Subendothelial Retention in Vascular Wall
W. Li (2014)
Enzyme Replacement Therapy in Orphan and Ultra-Orphan Diseases
D. Moore (2012)
Sphingomyelinase induces lipid microdomain formation in a fluid phosphatidylcholine/sphingomyelin membrane.
J. Holopainen (1998)
Receptor-mediated endocytosis: the intracellular journey of transferrin and its receptor.
A. Dautry-Varsat (1986)
Novel first-dose adverse drug reactions during a phase I trial of olipudase alfa (recombinant human acid sphingomyelinase) in adults with Niemann–Pick disease type B (acid sphingomyelinase deficiency)
M. Mcgovern (2016)
Enzymatic characterization of highly stable human alpha-galactosidase A displayed on magnetic particles
José Luis Corchero (2012)
Cell-Culture Models of the Blood–Brain Barrier
Y. He (2014)
In Drug Delivery, Shape Does Matter
S. Mitragotri (2008)
Neutralizing antibodies to therapeutic enzymes: considerations for testing, prevention and treatment
J. Wang (2008)
The tricyclic antidepressant desipramine causes proteolytic degradation of lysosomal sphingomyelinase in human fibroblasts.
R. Hurwitz (1994)
Jamming the endosomal system: lipid rafts and lysosomal storage diseases.
Kai Simons (2000)
pH and the recycling of transferrin during receptor-mediated endocytosis.
A. Dautry-Varsat (1983)
Lysosomal fusion and SNARE function are impaired by cholesterol accumulation in lysosomal storage disorders
A. Fraldi (2010)
Biological functionalization of drug delivery carriers to bypass size restrictions of receptor-mediated endocytosis independently from receptor targeting.
M. Ansar (2013)
Clarifying lysosomal storage diseases
Mark L. Schultz (2011)
Targeting endothelium and its dynamic caveolae for tissue-specific transcytosis in vivo: A pathway to overcome cell barriers to drug and gene delivery
D. Mcintosh (2002)
A practical chromogenic procedure for the detection of homozygotes and heterozygous carriers of Niemann-Pick disease.
A. Gal (1975)
Overendocytosis of gold nanoparticles increases autophagy and apoptosis in hypoxic human renal proximal tubular cells
F. Ding (2014)
Exosome Secretion Ameliorates Lysosomal Storage of Cholesterol in Niemann-Pick Type C Disease*
K. Strauss (2010)
Kahari, Matrix metalloproteinases in cancer: prognostic markers and therapeutic targets
P. Vihinen (2002)
The epidemiology of progressive intellectual and neurological deterioration in childhood
C. Verity (2009)
The effect of non‐ionic surfactant vesicle (niosome) entrapment on the absorption and distribution of methotrexate in mice
M. N. Azmin (1985)
Velaglucerase alfa: a new option for Gaucher disease treatment.
A. Zimran (2011)
Differential intra-endothelial delivery of polymer nanocarriers targeted to distinct PECAM-1 epitopes.
C. Garnacho (2008)
Lysosomal storage disease: revealing lysosomal function and physiology.
E. Parkinson-Lawrence (2010)
Nanomedicine and Drug Delivery Strategies for Treatment of Genetic Diseases
J. Hsu (2011)
Compact, Polyvalent Mannose Quantum Dots as Sensitive, Ratiometric FRET Probes for Multivalent Protein–Ligand Interactions
Y. Guo (2016)
Caveolae: from basic trafficking mechanisms to targeting transcytosis for tissue-specific drug and gene delivery in vivo.
J. Schnitzer (2001)
Regulated portals of entry into the cell
S. Conner (2003)
Genetic engineering of a lysosomal enzyme fusion protein for targeted delivery across the human blood‐brain barrier
R. Boado (2008)
A systematic analysis of peptide linker length and liposomal polyethylene glycol coating on cellular uptake of peptide-targeted liposomes.
J. F. Stefanick (2013)
Disease pathogenesis explained by basic science: lysosomal storage diseases as autophagocytic disorders.
A. Ballabio (2009)
Secrets of caveolae- and lipid raft-mediated endocytosis revealed by mammalian viruses.
L. Pelkmans (2005)
Acid Sphingomyelinase–Derived Ceramide Regulates ICAM-1 Function during T Cell Transmigration across Brain Endothelial Cells
M. A. Lopes Pinheiro (2016)
Clathrin-dependent and -independent internalization of plasma membrane sphingolipids initiates two Golgi targeting pathways
V. Puri (2001)
Targeting of protein kinase C-epsilon during Fcgamma receptor-dependent phagocytosis requires the epsilonC1B domain and phospholipase C-gamma1.
Keylon L. Cheeseman (2006)
Adenovirus triggers macropinocytosis and endosomal leakage together with its clathrin-mediated uptake
O. Meier (2002)
Structure and function of endothelial caveolae
R. Stan (2002)
Multi-walled carbon nanotubes induce human microvascular endothelial cellular effects in an alveolar-capillary co-culture with small airway epithelial cells
Brandi N. Snyder-Talkington (2013)
Reduction of nanoparticle avidity enhances the selectivity of vascular targeting and PET detection of pulmonary inflammation.
Blaine Zern (2013)
Recent advances in gene therapy for lysosomal storage disorders
D. Rastall (2015)
Recent advances in stealth coating of nanoparticle drug delivery systems.
Z. Amoozgar (2012)
Endocytosis and sorting of glycosphingolipids in sphingolipid storage disease.
D. Marks (2002)
New strategies for the treatment of lysosomal storage diseases (review).
G. Parenti (2013)
Gene delivery to differentiated neurotypic cells with RGD and HIV Tat peptide functionalized polymeric nanoparticles.
J. Suk (2006)
Pharmacokinetics and brain uptake in the rhesus monkey of a fusion protein of arylsulfatase a and a monoclonal antibody against the human insulin receptor
R. Boado (2013)
Cost-effectiveness of enzyme replacement therapy for Fabry disease
S. Rombach (2013)
Comparison of Five Peptide Vectors for Improved Brain Delivery of the Lysosomal Enzyme Arylsulfatase A
A. Böckenhoff (2014)
Clathrin-independent internalization and recycling
Q. Gong (2008)
Controlling ligand surface density optimizes nanoparticle binding to ICAM-1.
A. Fakhari (2011)
Lipid abnormalities in children with types A and B Niemann Pick disease.
M. Mcgovern (2004)
Folate receptor-mediated drug targeting: from therapeutics to diagnostics.
A. Hilgenbrink (2005)
Nanoparticle geometry and surface orientation influence mode of cellular uptake.
Heather L Herd (2013)
Intra- and trans-cellular delivery of enzymes by direct conjugation with non-multivalent anti-ICAM molecules.
Rasa Ghaffarian (2016)
Transport of nanocarriers across gastrointestinal epithelial cells by a new transcellular route induced by targeting ICAM-1.
Rasa Ghaffarian (2012)
Cell surface ceramide controls translocation of transferrin receptor to clathrin-coated pits.
Abo Bakr Abdel Shakor (2012)
Enzyme replacement therapy: conception, chaos and culmination.
R. Brady (2003)
Control of endothelial targeting and intracellular delivery of therapeutic enzymes by modulating the size and shape of ICAM-1-targeted carriers.
S. Muro (2008)
α-Galactosidase delivery using 30Kc19-human serum albumin nanoparticles for effective treatment of Fabry disease
Hong Jai Lee (2016)
Immunotargeting to ICAM-1 provides binding, internalization and lysosomal delivery of acid sphinomyelinase
S. Muro (2004)
Bright, Lysosomes: fusion and function
J. P. Luzio (2007)
Molecular pathophysiology of mucolipidosis type IV: pH dysregulation of the mucolipin-1 cation channel.
M. Raychowdhury (2004)
Virgin, Autophagy in immunity and inflammation
B. Levine (2011)
Mechanisms regulating endothelial cell barrier function.
T. Stevens (2000)
Soluble intercellular adhesion molecule-1 (sICAM-1): an overview.
A. Witkowska (2004)
Endothelial transcytosis in health and disease
M. Simionescu (2008)
A novel endocytic pathway induced by clustering endothelial ICAM-1 or PECAM-1
S. Muro (2003)
Ebolavirus Requires Acid Sphingomyelinase Activity and Plasma Membrane Sphingomyelin for Infection
M. Miller (2012)
Boosting Brain Uptake of a Therapeutic Antibody by Reducing Its Affinity for a Transcytosis Target
Y. J. Yu (2011)
CLN3 Loss Disturbs Membrane Microdomain Properties and Protein Transport in Brain Endothelial Cells
L. Tecedor (2013)
Biological aspects of ceramideenriched membrane domains
H. Grassme (2007)
Challenges in design and characterization of ligand-targeted drug delivery systems.
S. Muro (2012)
Human Rhinovirus Selectively Modulates Membranous and Soluble Forms of Its Intercellular Adhesion Molecule–1 (ICAM-1) Receptor to Promote Epithelial Cell Infectivity*
S. Whiteman (2003)
Targeted Polymeric Nanoparticles for Brain Delivery of High Molecular Weight Molecules in Lysosomal Storage Disorders
Marika Salvalaio (2016)
Elosulfase Alfa: A Review of Its Use in Patients with Mucopolysaccharidosis Type IVA (Morquio A Syndrome)
K. Lyseng-Williamson (2014)
Gene therapy for lysosomal storage diseases: the lessons and promise of animal models
N. M. Ellinwood (2004)
Matrix metalloproteinases: effectors of development and normal physiology.
T. Vu (2000)
Gene therapy for lysosomal storage diseases.
M. Sands (2006)
Thermo- and pH-responsive polymers in drug delivery.
D. Schmaljohann (2006)
Enzyme therapy. VI: Comparative in vivo fates and effects on lysosomal integrity of enzyme entrapped in negatively and positively charged liposomes.
L. D. Steger (1977)
The Hurler corrective factor. Purification and some properties.
R. Barton (1971)
Affinity and avidity in antibody-based tumor targeting.
Stephen I. Rudnick (2009)
A novel amino acid modification in sulfatases that is defective in multiple sulfatase deficiency
B. Schmidt (1995)
α-Mannosidase in Zinc-Deficient Rats: Possibility of Liposomal Therapy in Mannosidosis
H. M. Patel (1974)
The present and future of nanotechnology in human health care.
S. Sahoo (2007)
Long-circulating and target-specific nanoparticles: theory to practice.
S. Moghimi (2001)
ICAM-1 recycling in endothelial cells: a novel pathway for sustained intracellular delivery and prolonged effects of drugs.
S. Muro (2005)
Cysteamine therapy: a treatment for cystinosis, not a cure.
S. Cherqui (2012)
Ex Vivo Gene Therapy Using Patient iPSC-Derived NSCs Reverses Pathology in the Brain of a Homologous Mouse Model
Tagan A. Griffin (2015)
Profile of endothelial and leukocyte activation in fabry patients
T. DeGraba (2000)
[Lysosomal storage diseases--an overview].
A. Kloska (2011)
Stealth liposomes and long circulating nanoparticles: critical issues in pharmacokinetics, opsonization and protein-binding properties.
S. Moghimi (2003)
Gateways and tools for drug delivery: endocytic pathways and the cellular dynamics of cell penetrating peptides.
A. Jones (2008)
Autophagy in lysosomal storage disorders
A. Lieberman (2012)
The interaction of bacteria with mammalian cells.
S. Falkow (1992)
Targeted therapeutics and nanodevices for vascular drug delivery: Quo vadis?
V. Muzykantov (2011)
A general method for the introduction of enzymes, by means of immunoglobulin-coated liposomes, into lysosomes of deficient cells.
G. Weissmann (1975)
Torchilin, Lysosome-targeted octadecyl-rhodamine B-liposomes enhance lysosomal accumulation of 186 glucocerebrosidase in Gaucher's cells in vitro
R. Thekkedath (2013)
In vivo performance of polymer nanocarriers dually-targeted to epitopes of the same or different receptors.
Iason T. Papademetriou (2013)
Exploiting shape, cellular-hitchhiking and antibodies to target nanoparticles to lung endothelium: Synergy between physical, chemical and biological approaches.
Aaron C. Anselmo (2015)
Acidification of the endocytic and exocytic pathways.
I. Mellman (1986)
Cleavage of membrane‐associated ICAM‐1 from astrocytes: Involvement of a metalloprotease
P. Lyons (1998)
A Plant-Derived Recombinant Human Glucocerebrosidase Enzyme—A Preclinical and Phase I Investigation
D. Aviezer (2009)
Sodium dodecylsulphate induces a breach in the blood-brain barrier and enables a West Nile virus variant to penetrate into mouse brain
D. Kobiler (1989)
An emergency response team for membrane repair
P. L. McNeil (2005)
Endocytosis and molecular sorting.
I. Mellman (1996)
Dexamethasone-Mediated Up-Regulation of the Mannose Receptor Improves the Delivery of Recombinant Glucocerebrosidase to Gaucher Macrophages
Yunxiang Zhu (2004)
Control of intracellular trafficking of ICAM-1-targeted nanocarriers by endothelial Na+/H+ exchanger proteins.
S. Muro (2006)
Different Contributions of Clathrin- and Caveolae-Mediated Endocytosis of Vascular Endothelial Cadherin to Lipopolysaccharide-Induced Vascular Hyperpermeability
Y. Zhang (2014)
Bello-Reuss, Kidney., in Intercelluar Signaling in Development and Disease
W. J. Arendshorst (2011)
Transferrin receptor (TfR) trafficking determines brain uptake of TfR antibody affinity variants
N. Bien-ly (2014)
Action of BTN1, the yeast orthologue of the gene mutated in Batten disease
D. Pearce (1999)
V. Torchilin (2006)
Glycolipid Trafficking in Drosophila Undergoes Pathway Switching in Response to Aberrant Cholesterol Levels
R. Hortsch (2010)
Recent developments in lipid-based pharmaceutical nanocarriers.
T. Musacchio (2011)
Soluble intercellular adhesion molecule-1 inhibits MHC-restricted specific T cell/tumor interaction.
J. Becker (1993)
Amantadine and dansylcadaverine inhibit vesicular stomatitis virus uptake and receptor-mediated endocytosis of alpha 2-macroglobulin.
R. Schlegel (1982)
Targeting, Endocytosis, and Lysosomal Delivery of Active Enzymes to Model Human Neurons by ICAM-1-Targeted Nanocarriers
Janet Hsu (2014)
Soluble ICAM-1: a marker of vascular inflammation and lifestyle.
A. Witkowska (2005)
How Carrier Size and Valency Modulate Receptor-Mediated Signaling: Understanding the Link between Binding and Endocytosis of ICAM-1-Targeted Carriers.
Daniel Serrano (2016)
Regulated secretion of conventional lysosomes.
N. Andrews (2000)
Nanoparticles: a boon to drug delivery, therapeutics, diagnostics and imaging.
S. Parveen (2012)
Prevention of lysosomal storage in Tay-Sachs mice treated with N-butyldeoxynojirimycin.
F. Platt (1997)
Targeting of lysosomes by liposomes modified with octadecyl-rhodamine B
Alexander Koshkaryev (2011)
Comparative binding, endocytosis, and biodistribution of antibodies and antibody-coated carriers for targeted delivery of lysosomal enzymes to ICAM-1 versus transferrin receptor
J. Papademetriou (2012)
ROLE OF ICAM-1-MEDIATED ENDOCYTOSIS IN ENDOTHELIAL FUNCTION AND IMPLICATIONS FOR CARRIER-ASSISTED DRUG DELIVERY
D. Serrano (2014)
Efficacy of 2-Hydroxypropyl-β-cyclodextrin in Niemann-Pick Disease Type C Model Mice and Its Pharmacokinetic Analysis in a Patient with the Disease.
Y. Tanaka (2015)
pH-responsive polysaccharide-based polyelectrolyte complexes as nanocarriers for lysosomal delivery of therapeutic proteins.
M. I. Giannotti (2011)
Uptake and distribution of placental glucocerebrosidase in rat hepatic cells and effects of sequential deglycosylation.
F. S. Furbish (1981)
Enzyme replacement with liposomes containing beta-galactosidase from Charonia lumpas in murine globoid cell leukodystrophy (twitcher).
F. Umezawa (1985)
Lysosomes and Nanotherapeutics: Diseases, Treatments, and Side Effects
R. L. Manthe (2014)
Pathways of clathrin-independent endocytosis
S. Mayor (2007)
Optimizing endothelial targeting by modulating the antibody density and particle concentration of anti-ICAM coated carriers.
A. J. Calderon (2011)
Extracellular matrix remodeling and matrix metalloproteinases in the vascular wall during aging and in pathological conditions.
M. Jacob (2003)
Matrix metalloproteinase 9 (MMP-9/gelatinase B) proteolytically cleaves ICAM-1 and participates in tumor cell resistance to natural killer cell-mediated cytotoxicity
E. Fiore (2002)
A transmigratory cup in leukocyte diapedesis both through individual vascular endothelial cells and between them
C. Carman (2004)
Mannose 6-Phosphate Receptor-mediated Uptake Is Defective in Acid Sphingomyelinase-deficient Macrophages
Rajwinder Dhami (2004)
Transepithelial and endothelial transport of poly (amidoamine) dendrimers.
Kelly M. Kitchens (2005)
Clathrin-Mediated Endocytosis Is Impaired in Type A–B Niemann–Pick Disease Model Cells and Can Be Restored by ICAM-1-Mediated Enzyme Replacement
J. Rappaport (2014)
Cellular uptake and lysosomal delivery of galactocerebrosidase tagged with the HIV Tat protein transduction domain
Xianyang Zhang (2008)
Altered Clathrin-Independent Endocytosis in Type A Niemann-Pick Disease Cells and Rescue by ICAM-1-Targeted Enzyme Delivery.
J. Rappaport (2015)
Abnormal mannose-6-phosphate receptor trafficking impairs recombinant alpha-glucosidase uptake in Pompe disease fibroblasts
M. Cardone (2008)
Lysosomal localization of enzyme-containing liposomes injected into rats.
G. Gregoriadis (1972)
Inflammatory and cytotoxic responses of an alveolar-capillary coculture model to silica nanoparticles: Comparison with conventional monocultures
Jennifer Y Kasper (2010)
Delivery of fungal beta-galactosidase to rat brain by means of liposomes.
G. Takada (1982)
Enhanced endothelial delivery and biochemical effects of α-galactosidase by ICAM-1-targeted nanocarriers for Fabry disease.
Janet Hsu (2011)
A synthetic chaperone corrects the trafficking defect and disease phenotype in a protein misfolding disorder
G. Yam (2005)
Comparative biology of intracellular parasitism.
J. Moulder (1985)
Gene therapy for lysosomal storage disorders
A. Gritti (2011)
Effect of flow on endothelial endocytosis of nanocarriers targeted to ICAM-1.
T. Bhowmick (2012)
Lysosomal Transport Disorders
G. Mancini (2006)
Therapy of adult Gaucher disease.
Joerg Schmitz (2007)
Enhanced intracellular stability and efficacy of PEG modified dextranase in the treatment of a model storage disorder.
S. Mumtaz (1994)
Ceria nanoparticles stabilized by organic surface coatings activate the lysosome-autophagy system and enhance autophagic clearance.
W. Song (2014)