α-Galactosidase delivery using 30Kc19-human serum albumin nanoparticles for effective treatment of Fabry disease

Lee, Hong Jai; Park, Hee Ho; Sohn, Youngsoo; Ryu, Jina; Park, Jae Young; Rhee, Won Jong; Park, Tai Hyun

doi:10.1007/s00253-016-7689-z

Cited by 24 publications

(11 citation statements)

References 26 publications

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“…Trimethyl chitosan nanoparticles tested in cellular models showed enzyme dissociation from the nanoparticle at lysosomal pH [411]. Protein-based nanoparticles (albumin and silkworm 30Kc19 protein) have rendered enhanced uptake and stability of α-galactosidase (deficient in Fabry disease) in patient fibroblast cultures [412]. …”

Section: Additional Approaches Toward the Optimization Of Lysosomamentioning

confidence: 99%

Lysosomal enzyme replacement therapies: Historical development, clinical outcomes, and future perspectives

Solomon

Muro

2017

Advanced Drug Delivery Reviews

113

110

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Lysosomes and lysosomal enzymes play a central role in numerous cellular processes, including cellular nutrition, recycling, signaling, defense, and cell death. Genetic deficiencies of lysosomal components, most commonly enzymes, are known as “lysosomal storage disorders” or “lysosomal diseases” (LDs) and lead to lysosomal dysfunction. LDs broadly affect peripheral organs and the central nervous system (CNS), debilitating patients and frequently causing fatality. Among other approaches, enzyme replacement therapy (ERT) has advanced to the clinic and represents a beneficial strategy for 8 out of the 50–60 known LDs. However, despite its value, current ERT suffers from several shortcomings, including various side effects, development of “resistance”, and suboptimal delivery throughout the body, particularly to the CNS, lowering the therapeutic outcome and precluding the use of this strategy for a majority of LDs. This review offers an overview of the biomedical causes of LDs, their socio-medical relevance, treatment modalities and caveats, experimental alternatives, and future treatment perspectives.

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Section: Additional Approaches Toward the Optimization Of Lysosomamentioning

confidence: 99%

Lysosomal enzyme replacement therapies: Historical development, clinical outcomes, and future perspectives

Solomon

Muro

2017

Advanced Drug Delivery Reviews

113

110

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“…Particle size was found to be dependent on the protein concentration, volume, pH, and temperature of the desolvating agents for the volume of protein solution. Lee et al encapsulated and delivered α-galactosidase (α-gal) into HSA and recombinant 30Kc19 protein nanoparticles [ 87 ]. 30Kc19 is a silkworm protein with cell-penetrating and enzyme-stabilizing effects.…”

Section: Methods For Producing Protein Nanoparticlesmentioning

confidence: 99%

“…Using the 30Kc19 protein nanoparticles, 30 to 50% of β-gal was released within 24 h and continuous release up to 60% was observed. They also generated nanoparticles containing α-galactosidase (α-gal) using 30Kc19 and HSA, and successfully delivered them to cells [ 87 ]. As the wt% of 30Kc19 protein increased from 0 to 70%, the size of nanoparticles increased from 230 nm to 310 nm.…”

Section: Types Of Proteins Used To Produce Protein Nanoparticlesmentioning

confidence: 99%

“…The advantage of Ontak is that it has targeted T-cell specificity and lysosomal escape property. A possible reason for the presence of only two protein nano-drugs in the nanomedicines approved by the FDA list is that albumin has its own endocytosis route mediated by albumin receptor gp60, located at caveolae [ 87 ]. With regard to cells that lack or have limited caveolae, additional ligands are required to efficiently deliver albumin nanoparticles loaded with therapeutics into target cells.…”

Section: Fda Approval Of Protein Nano-drugs For Medical Purposesmentioning

confidence: 99%

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Protein-Based Nanoparticles as Drug Delivery Systems

et al. 2020

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Nanoparticles have been extensively used as carriers for the delivery of chemicals and biomolecular drugs, such as anticancer drugs and therapeutic proteins. Natural biomolecules, such as proteins, are an attractive alternative to synthetic polymers commonly used in nanoparticle formulation because of their safety. In general, protein nanoparticles offer many advantages, such as biocompatibility and biodegradability. Moreover, the preparation of protein nanoparticles and the corresponding encapsulation process involved mild conditions without the use of toxic chemicals or organic solvents. Protein nanoparticles can be generated using proteins, such as fibroins, albumin, gelatin, gliadine, legumin, 30Kc19, lipoprotein, and ferritin proteins, and are prepared through emulsion, electrospray, and desolvation methods. This review introduces the proteins used and methods used in generating protein nanoparticles and compares the corresponding advantages and disadvantages of each.

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“…Using primary cultured fibroblast, 30Kc19-HSA NPs led to enhanced cellular uptake of the carried protein. The α-galactosidase carried by these NPs had improved globotriaosylceramide degradation in the fibroblasts (Lee et al, 2016).…”

Section: Np-based Delivery Of Therapeutic Molecules For the Treatmentmentioning

confidence: 99%

CNS-Targeting Therapies for Lysosomal Storage Diseases: Current Advances and Challenges

Edelmann

Maegawa

2020

Front. Mol. Biosci.

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During the past decades, several therapeutic approaches have been developed and made rapidly available for many patients afflicted with lysosomal storage disorders (LSDs), inborn organelle disorders with broad clinical manifestations secondary to the progressive accumulation of undegraded macromolecules within lysosomes. These conditions are individually rare, but, collectively, their incidence ranges from 1 in 2,315 to 7,700 live-births. Most LSDs are manifested by neurological symptoms or signs, including developmental delay, seizures, acroparesthesia, motor weakness, and extrapyramidal signs. The chronic and later-onset clinical forms are at one end of the continuum spectrum and are characterized by a subtle and slow progression of neurological symptoms. Due to its inherent physiological properties, unfortunately, the blood-brain barrier (BBB) constitutes a significant obstacle for current and upcoming therapies to achieve the central nervous system (CNS) and treat neurological problems so prevalent in these conditions. To circumvent this limitation, several strategies have been developed to make the therapeutic agent achieve the CNS. This narrative will provide an overview of current therapeutic strategies under development to permeate the BBB, and address and unmet need for treatment of the progressive neurological manifestations, which are so prevalent in these inherited lysosomal disorders.

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α-Galactosidase delivery using 30Kc19-human serum albumin nanoparticles for effective treatment of Fabry disease

Cited by 24 publications

References 26 publications

Lysosomal enzyme replacement therapies: Historical development, clinical outcomes, and future perspectives

Lysosomal enzyme replacement therapies: Historical development, clinical outcomes, and future perspectives

Protein-Based Nanoparticles as Drug Delivery Systems

CNS-Targeting Therapies for Lysosomal Storage Diseases: Current Advances and Challenges

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