Liposomal formulations for treating lysosomal storage disorders

Tomsen-Melero, Judit; Merlo-Mas, Josep; Carreño, Aida; Sala, Santiago; Córdoba, Alba; Veciana, Jaume; González‐Mira, Elisabet; Ventosa, Nora

doi:10.1016/j.addr.2022.114531

Cited by 8 publications

(2 citation statements)

References 173 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…17 To obtain nanoliposomes with desirable size distributions, the high-pressure microfluidizer has been widely applied in large-scale liposome preparation, which relies on the combined force of high-velocity impact, high-frequency vibration, intense shear, and cavi-tation. 18 Besides, surface modification of liposomes by antibodies, peptides, and polysaccharides alters the bio-distribution of drugs, further increasing drug accumulation at the lesion site instead of non-specific tissues. 19,20 HAP-1 peptide can efficiently and selectively facilitate protein internalization into synovial cells and thus can serve as a targeting ligand for FLS-targeting nanosystems for RA management.…”

Section: Introductionmentioning

confidence: 99%

Surface-decorated nanoliposomal leonurine targets activated fibroblast-like synoviocytes for efficient rheumatoid arthritis therapy

Meng,

Song,

Tang

et al. 2023

Biomater. Sci.

View full text Add to dashboard Cite

show abstract

Section: Introductionmentioning

confidence: 99%

Surface-decorated nanoliposomal leonurine targets activated fibroblast-like synoviocytes for efficient rheumatoid arthritis therapy

Meng,

Song,

Tang

et al. 2023

Biomater. Sci.

View full text Add to dashboard Cite

show abstract

“…These nanosystems can also display targeting elements [ 11 , 16 ]. Nanocarriers under investigation for lysosomal ERT include exosomes and extracellular vesicles [ 17 ], liposomes and other lipid-based systems [ 18 ], and polymer-based nanoparticles (NPs) [ 19 ]. Among the latter class, NPs made from poly(lactic-co-glycolic acid) or PLGA have drawn attention for lysosomal ERT because of various advantages.…”

Section: Introductionmentioning

confidence: 99%

Role of the Lactide:Glycolide Ratio in PLGA Nanoparticle Stability and Release under Lysosomal Conditions for Enzyme Replacement Therapy of Lysosomal Storage Disorders

del Moral,

Loeck,

Muntimadugu

et al. 2023

JFB

View full text Add to dashboard Cite

Prior studies demonstrated that encapsulation in poly(lactic-co-glycolic acid) (PLGA) nanoparticles (NPs) enhanced the delivery of enzymes used for replacement therapy (ERT) of lysosomal storage disorders (LSDs). This study examined how the copolymer lactide:glycolide ratio impacts encapsulation, physicochemical characteristics, stability, and release under lysosomal conditions. Hyaluronidase, deficient in mucopolysaccharidosis IX, was encapsulated in NPs synthesized using 50:50, 60:40, or 75:25 lactide:glycolide copolymers. All NPs had diameters compatible with cellular transport (≤168 nm) and polydispersity indexes (≤0.16) and ζ-potentials (≤−35 mV) compatible with colloidal stability. Yet, their encapsulation efficiency varied, with 75:25 NPs and 60:40 NPs having the lowest and highest EE, respectively (15% vs. 28%). Under lysosomal conditions, the 50:50 copolymer degraded fastest (41% in 1 week), as expected, and the presence of a targeting antibody coat did not alter this result. Additionally, 60:40 NPs destabilized fastest (<1 week) because of their smaller diameter, and 75:25 NPs did not destabilize in 4 weeks. All formulations presented burst release under lysosomal conditions (56–78% of the original load within 30 min), with 50:50 and 60:40 NPs releasing an additional small fraction after week 1. This provided 4 weeks of sustained catalytic activity, sufficient to fully degrade a substrate. Altogether, the 60:40 NP formulation is preferred given its higher EE, and 50:50 NPs represent a valid alternative, while the highest stability of 75:25 NPs may impair lysosomes. These results can guide future studies aiming to translate PLGA NP-based ERT for this and other LSDs.

show abstract