Polymer-based drug delivery systems under investigation for enzyme replacement and other therapies of lysosomal storage disorders

Placci, Marina; Giannotti, Marina I.; Muro, Silvia

doi:10.1016/j.addr.2022.114683

Cited by 13 publications

(5 citation statements)

References 506 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…The problems limit their future preclinical tests. [65,66] The unique stimuli presented in cytosol or organelles but not other intracellular or extracellular spaces are a higher temperature (e.g., 38-48 °C) [61] than the average body temperature (i.e., 37 °C) and a higher pH (e.g., pH 8) than the normal physiological pH (i.e., pH 7.4) in mitochondria. These stimuli could weaken the interaction between the drug and carrier, triggering drug re-lease in the organelle.…”

Section: 𝛾-Glutamylamidementioning

confidence: 99%

Beyond Nanoparticle‐Based Intracellular Drug Delivery: Cytosol/Organelle‐Targeted Drug Release and Therapeutic Synergism

Cho,

Huh,

Shim

et al. 2024

Macromolecular Bioscience

View full text Add to dashboard Cite

Nanoparticle (NP)‐based drug delivery systems have been conceived to solve poor water‐solubility and chemical/physical instability, and their purpose expanded to target specific sites for maximizing therapeutic effects and minimizing unwanted events of payloads. Targeted sites have also been narrowed from organs/tissues and cells to cytosol/organelles. Beyond specific site targeting, the particular release of payloads at the target sites is growing in importance. This review overviews various issues and their general strategies during multiple steps, from the preparation of drug‐loaded NPs to their drug release at the target cytosol/organelles. In particular, this review focuses on current strategies for “first” delivery and “later” release of drugs to the cytosol or organelles of interest using specific stimuli in the target sites. Recognizing or distinguishing the presence/absence of stimuli or their differences in concentration/level/activity in one place from those in another has been applied to stimuli‐triggered release via bond cleavage or nanostructural transition. In addition, future directions on understanding the intracellular balance of stimuli and their counter‐stimuli are demonstrated to synergize the therapeutic effects of payloads released from stimuli‐sensitive NPs.This article is protected by copyright. All rights reserved

show abstract

Section: 𝛾-Glutamylamidementioning

confidence: 99%

Beyond Nanoparticle‐Based Intracellular Drug Delivery: Cytosol/Organelle‐Targeted Drug Release and Therapeutic Synergism

Cho,

Huh,

Shim

et al. 2024

Macromolecular Bioscience

View full text Add to dashboard Cite

show abstract

“…108,141 The purpose of this encapsulation is likely to protect the enzyme aggregates during transport and delivery, ensuring their stability and targeted release at the desired site, which is the CNS. 108,141 The addition of brain-targeting peptides (Ang2, g7, or Tf2) further enhances the specificity of the delivery system, ensuring that the encapsulated enzyme reaches the intended target within the CNS. 108 Del Moral et al examined the PLGA NP technique, evaluating the efficacy of NPs based on their lactide and glycolide composition.…”

Section: Enzyme Replacement Therapymentioning

confidence: 99%

“…In this novel approach, the researchers utilized CLEAs of the enzyme GALC 108 . These CLEAs were encapsulated within PLGA NPs 108,141 . The purpose of this encapsulation is likely to protect the enzyme aggregates during transport and delivery, ensuring their stability and targeted release at the desired site, which is the CNS 108,141 .…”

Section: Treatmentmentioning

confidence: 99%

From pathological mechanisms in Krabbe disease to cutting‐edge therapy: A comprehensive review

Ketata,

Ellouz

2024

Neuropathology

View full text Add to dashboard Cite

Since its initial documentation by Knud Krabbe in 1916, numerous studies have scrutinized the characteristics of Krabbe disease (KD) until the identification of the mutation in the GALC gene. In alignment with that, we investigated the natural history of KD spanning eight decades to gain a deeper understanding of the evolutionary trajectory of its mechanisms. Through our comprehensive analysis, we unearthed additional novel elements in molecular biology involving the micropathological mechanism of the disease. This review offers an updated perspective on the metabolic disorder that defines KD. Recently, extracellular vesicles (EVs), autophagy impairment, and α‐synuclein have emerged as pivotal players in the neuropathological processes. EVs might serve as a cellular mechanism to avoid or alleviate the detrimental impacts of excessive toxic psychosine levels, and extracting EVs could contribute to synapse dysfunction. Autophagy impairment was found to be independent of psychosine and reliant on AKT and B‐cell lymphoma 2. Additionally, α‐synuclein has been recognized for inducing cellular death and dysfunction in common biological pathways. Our objective is to assess the effectiveness of advanced therapies in addressing this particular condition. While hematopoietic stem cells have been a primary treatment, its administration proves challenging, particularly in the presymptomatic phase. In this review, we have compiled information from over 10 therapy trials, comparing them based on their benefits and disadvantage.

show abstract

“…It is feasible to improve disease diagnosis and treatment specificity using designed nanoparticles [1][2][3]. For instance nanoparticles represent promising formulations that may ameliorate the stability and solubility of encapsulated drugs, improve transport across membranes and affect circulation times to increase safety and efficacy of nano-based medicines [4,5]. Furthermore, novel nanomaterials (e.g.…”

Section: Introductionmentioning

confidence: 99%

Impact of mechanical cues on key cell functions and cell-nanoparticle interactions

Elblová,

Lunova,

Dejneka

et al. 2024

Discover Nano

View full text Add to dashboard Cite

In recent years, it has been recognized that mechanical forces play an important regulative role in living organisms and possess a direct impact on crucial cell functions, ranging from cell growth to maintenance of tissue homeostasis. Advancements in mechanobiology have revealed the profound impact of mechanical signals on diverse cellular responses that are cell type specific. Notably, numerous studies have elucidated the pivotal role of different mechanical cues as regulatory factors influencing various cellular processes, including cell spreading, locomotion, differentiation, and proliferation. Given these insights, it is unsurprising that the responses of cells regulated by physical forces are intricately linked to the modulation of nanoparticle uptake kinetics and processing. This complex interplay underscores the significance of understanding the mechanical microenvironment in shaping cellular behaviors and, consequently, influencing how cells interact with and process nanoparticles. Nevertheless, our knowledge on how localized physical forces affect the internalization and processing of nanoparticles by cells remains rather limited. A significant gap exists in the literature concerning a systematic analysis of how mechanical cues might bias the interactions between nanoparticles and cells. Hence, our aim in this review is to provide a comprehensive and critical analysis of the existing knowledge regarding the influence of mechanical cues on the complicated dynamics of cell-nanoparticle interactions. By addressing this gap, we would like to contribute to a detailed understanding of the role that mechanical forces play in shaping the complex interplay between cells and nanoparticles.

show abstract

Polymer-based drug delivery systems under investigation for enzyme replacement and other therapies of lysosomal storage disorders

Cited by 13 publications

References 506 publications

Beyond Nanoparticle‐Based Intracellular Drug Delivery: Cytosol/Organelle‐Targeted Drug Release and Therapeutic Synergism

Beyond Nanoparticle‐Based Intracellular Drug Delivery: Cytosol/Organelle‐Targeted Drug Release and Therapeutic Synergism

From pathological mechanisms in Krabbe disease to cutting‐edge therapy: A comprehensive review

Impact of mechanical cues on key cell functions and cell-nanoparticle interactions

Contact Info

Product

Resources

About