Ocular drug delivery is constrained by anatomical barriers, continuous clearance, and compartment-specific homeostasis, which together reduce bioavailability and shorten exposure at target tissues. This review focuses on natural polysaccharide microspheres (NPMs), defined as micron-scale particles whose primary matrix or essential crosslinking framework is a natural polysaccharide or its derivative. Rather than treating polysaccharides as a uniform class, we examine how molecular weight, charge, viscosity, and crosslinking behavior affect fabrication, particle properties, ocular retention, and release. We also compare their route-specific uses and limitations with those of other sustained-release systems. Furthermore, the biological functions of polysaccharide excipients and the concept of “drug-excipient integration” are critically analyzed. Finally, this review evaluates the biosafety, manufacturing processes (including sterilization and quality control), regulatory considerations, patent landscape, clinical evidence, and translational requirements of NPMs, providing guidance for the development of next-generation safe and effective ocular drug delivery platforms.
Vitiligo is characterized by a complex pathogenesis involving multiple factors, necessitating therapeutic strategies that simultaneously achieve immune regulation, melanocyte regeneration, and melanosome transfer modulation. Here, we designed a Janus microneedle (Janus MN) based on a core-shell bilayer structure, inspired by the melanin metabolism pathway and the stratified architecture of the skin. The shell of the MN was loaded with a complex of hypericin-assembled nanoparticles (HY-NPs) and astragalus polysaccharides, which preferentially dissolve in the epidermal hair follicle region and basal layer. This intervention ameliorated the microenvironment by scavenging reactive oxygen species and inhibiting CD8+ T-cell-mediated immune attacks on melanocytes for repair, while simultaneously activating the MAPK pathway to enhance melanin synthesis in melanocytes for regeneration. The MN core, encapsulated with stabilized trypsin in a hyaluronic acid matrix, targeted the upper epidermal keratinocytes. It activated the PAR2 signaling pathway to enhance melanosome transfer and phagocytosis, thereby promoting visible pigmentation. It integrated dual spatially heterogeneous targets within the epidermis and hair follicles for the first time, achieving multidimensional synergistic intervention through a sequence of "repair, regeneration, and transfer". Our study provides a novel, spatiotemporally sequential drug delivery strategy for the precise treatment of vitiligo.
Nanomedicines have been constructed to enhance the efficacy and safety of traditional anti-cancer drugs. However, the clinical translation of these nanomedicines was hindered by the complexity of carrier materials and functional integration. In this study, a carrier-free nanomedicine composed of doxorubicin (DOX), Cu2+ and siRNA of GPX4 was constructed for combination therapy of tumors. DOX, Cu2+ and siRNA were assembled through multiple forces and decorated with hyaluronic acid (HA) to prepare tumor targeted carrier-free nanomedicines (HDCS). The HA coating on the surface of HDCS ensured specific tumor targeting, and the released drugs triggered by acidity could eliminate tumor cells. The nanomedicine showed significant anti-cancer efficiency and quite reduced side effects in the breast cancer model when compared with DOX solution. HDCS treatment led to significant accelerated production of ROS, reduced GPX4 expression, and sustained decrease in GSH at tumor sites, ultimately forming a powerful chemodynamic effect enhancing circuit. The HDCS provides an effective strategy for tumor combination therapy with good clinical translation and application potential.
The stimulator of interferon genes (STING) pathway plays a critical role in bridging innate and adaptive antitumor immunity, representing a promising target in cancer immunotherapy. However, the clinical application of STING agonists is limited by poor pharmacokinetics, low cytosolic delivery efficiency, and immune-related adverse effects. To address these challenges, we developed STING polyproagonist nanoparticles (named GA+S@SR) by self-assembly of an amphiphilic diblock copolymer, P(OEGMA-co-GAMA)-b-PSSRMA, combined with POEGMA-b-P(DEAEMA-co-BMA). The galactose (GA) moieties enable targeted delivery to glucose transporter 1 (GLUT1) on tumor cells, facilitating cellular internalization. The P(DEAEMA-co-BMA) segments promote endosomal escape, followed by the release of the disulfide-linked SR-717 in the cytosol under reducing conditions. This leads to robust activation of the STING pathway, resulting in dendritic cell maturation, enhanced T-cell infiltration, and potent antitumor immunity. Furthermore, when combined with an αPD-L1, this polyproagonist synergistically enhances the efficacy of immune checkpoint blockade, effectively inhibiting primary and distant tumors by counteracting immune evasion. This study highlights the potential of STING polyproagonists in achieving effective cytosolic delivery of STING agonists to boost antitumor immunity and overcome current limitations associated with STING immunotherapy.
With low five-year survival estimates, poor prognosis, and high recurrence probabilities, glioma is considered one of the most intractable malignant tumors. Despite the discovery of lymphatic vascular system and immune system in the central nervous system (CNS), immune checkpoint blockade therapeutics, such as programmed death ligand 1 (PDL1, also called B7H1 or CD274) antibodies, are prevented from the CNS and glioma sites due to the existence of biological barriers including the blood-brain barrier (BBB) and blood-brain tumor barrier (BBTB). Herein, we constructed a BBB/BBTB-crossing recombinant antibody by fusing the PDL1 antibody (αPDL1) and the targeting moiety RAP22 peptide (RAP22) through a matrix metalloproteinase 2 (MMP2)-responsive cleavable linker, abbreviated as αPDL1-mRAP22. Not only was αPDL1-mRAP22 able to block PD1/PDL1 pathway, reduce T cell apoptosis, enhance T cell killing ability towards glioma cells in vitro, but also it showed higher accumulation in the glioma site, prolonged survival time, and potent immune responses as well as synergistic effects with temozolomide (TMZ) in vivo, offering a novel strategy for glioma immunotherapy.
Over the past decade, accumulating evidence supporting the efficacy of probiotics in gut microbiota modulation has spurred interest in their therapeutic potential for inflammatory bowel disease (IBD), a chronic intestinal disorder for which safer and more sustainable alternatives to conventional immunosuppressive and biologic treatments are urgently needed. Probiotics represent a promising therapeutic strategy due to their favorable biosafety profile and compatibility with daily health practices. Mechanistically, probiotics mitigate intestinal inflammation by suppressing pathogenic bacteria, modulating gut immunity and regulating microbial metabolites. However, their application is limited by low viability and poor intestinal colonization following oral administration. To address these challenges, advanced delivery strategies, including physical encapsulation, biohybrid nanocoating and chemical modifications, have been developed to enhance probiotic survival and targeted delivery. Despite these advances, current probiotic delivery systems face limitations, such as the impairment of microbial activity by surface modifications, deficiency in formulating intestinal digestion resistance strategies, lack of strategies for active colonization, and incomplete understanding of therapeutic mechanisms. By addressing these barriers, targeted and sustained research into innovative probiotic delivery systems may yield novel therapeutic approaches for IBD. This review indicates that relying solely on physical encapsulation or simple coating can no longer meet therapeutic demands. Future efforts must shift toward a functional synergy direction, developing multifunctional systems that protect probiotic viability without inhibiting their metabolic and physiological functions while integrating active colonization and targeted release. Furthermore, the review emphasizes the current incompleteness in understanding the therapeutic mechanisms of probiotics and calls for embedding mechanistic studies into the rational design of delivery systems. These insights provide a pathway for the field to move from determining whether live probiotics can be delivered to exploring how to achieve efficient, safe and functionally intact oral probiotic therapy.
The rising threat of antibiotic resistance severely compromises the treatment of bacterial pneumonia, underscoring the urgent need for innovative and comprehensive therapeutic strategies. Here, we developed an innovative theranostic nanoplatform (Dex/BTGd) through the coordinated assembly of tetracycline, gadolinium ion (Gd3+) and baicalin, which acts synergistically to combat infection, enable non-invasive magnetic resonance imaging (MRI), and regulate the lung microenvironment. Dex/BTGd demonstrated potent antibacterial activity against clinically relevant pathogens, effectively disrupting biofilms and overcoming tetracycline resistance. The incorporated Gd3+ allowed targeted visualization of pulmonary infection sites by MRI, facilitating timely disease assessment. Beyond its direct antibacterial effects, the nanoplatform modulated the inflammatory microenvironment by scavenging reactive oxygen species, promoting macrophage polarization from the pro-inflammatory to the anti-inflammatory phenotype, and suppressing profibrotic transforming growth factor-β signaling. In a murine model of tetracycline-resistant Pseudomonas aeruginosa pneumonia, Dex/BTGd significantly reduced bacterial load, alleviated pulmonary edema and fibrosis, and enabled targeted MRI visualization of infected lesions. This integrated strategy of pathogen clearance, microenvironment modulation, and tissue repair offers a multifaceted solution to drug-resistant bacterial pneumonia, providing a promising blueprint for next-generation anti-infective systems.
Following injury, prostaglandin E2 (PGE2) drives intestinal epithelial repair by inducing revival stem cells (RSCs), which compensate for the loss of homeostatic Lgr5+ stem cells. Using intestinal organoid models, we demonstrate that melatonin potentiates the PGE2- or damage-induced RSC emergence by rewiring cellular plasticity toward a fetal-like state and sustaining pro-regenerative YAP activity, thereby enhancing overall repair capacity. To translate this finding into a therapeutic application, we developed a biohybrid heterospheroid (Mel-HS) by combining melatonin-loaded poly(lactic-co-glycolic acid) microspheres with 3D-cultured mesenchymal stem cells (MSCs), which serve as a PGE2 source. We confirmed that this biohybrid construct preserves the paracrine capacity of MSCs to secrete PGE2. Notably, Mel-HS demonstrates superior in vivo retention compared with naive 3D-MSCs, underscoring the cytoprotective effect of encapsulated melatonin in enhancing MSC viability. Furthermore, Mel-HS promoted robust RSC induction while simultaneously providing protection against inflammatory- and oxidative insults in vitro. In a colitis model, Mel-HS accelerated mucosal healing through the dual mechanisms-immunomodulation and enhanced RSC-driven repair-resulting in marked clinical improvement. Collectively, our findings highlight the therapeutic potential of enhancing endogenous regeneration with melatonin and MSCs, establishing a promising framework for next-generation biohybrid cell therapeutics in inflammatory bowel disease management.
Conventional therapy for ulcerative colitis (UC) is often limited by insufficient colonic targeting, short local retention and poor cellular drug uptake at lesion sites. Here, we develop a biomimetic colon-targeted delivery system based on an opposite pH-responsive "gating" strategy. Chrysanthemum sporopollenin (spo) microcapsules with a characteristic spiny architecture serve as the core carrier. Spo exhibits acid-induced contraction and alkali-induced expansion, with germinal apertures opening progressively as pH increases. In contrast, chitosan-butyrate complex (CBC) swells into a gel under acidic conditions but contracts and precipitates in alkaline environments. After drug loading into the spo, surface coating with CBC seals the germinal apertures, constructing an intelligent gate. In gastric fluid, the CBC layer gels and blocks apertures to prevent premature drug release. In intestinal fluid, CBC contracts and precipitates to open the gate; meanwhile, spo expands to further widen germinal apertures and facilitate drug release. The spiny morphology of spo, combined with the mucoadhesive properties of chitosan and active targeting of butyrate, collectively enhances intestinal adhesion and retention, enabling precise colonic drug release and accumulation. Mesalazine is formulated into liposomes to improve aqueous solubility and stability, which enhances cellular uptake and bioavailability, thereby exerting synergistic anti-inflammatory effects with butyric acid at inflamed sites. The chrysanthemum sporopollenin-based gated microcapsules exhibit favorable pH-responsive release, enhanced mucoadhesion and potent synergistic anti-inflammatory activity. This work provides a promising multifunctional targeted delivery strategy for UC therapy and establishes a novel, versatile design concept termed the opposite pH-responsive dual-gating mechanism, which supports the development of oral colon-targeted carriers capable of navigating complex gastrointestinal environments.
Cytoreductive surgery is the treatment protocol for colorectal cancer. Nonetheless, a major medical challenge remains to fully eliminate malignant tumor cells, along with a number of complications such as peritoneal adhesion and tumor peritoneal metastasis. The occurrence of peritoneal adhesions compromises not only the ability to do subsequent surgery, but also the efficacy of adjunct chemotherapy. More and more evidence suggest that the process of mesothelial-mesenchymal transition (MMT) influenced by transforming growth factor-β1 (TGF-β1) has a role to play in these disturbances, therefore making TGF-β1 a viable target for therapy. This study has designed a hydrogel-based physical barrier drug delivery system loaded with RNA interference technology, designated as FC@MT. The 5-fluorouracil (5-FU), which is known for its antitumor effects, was firmly linked to the FCGCM hydrogel matrix through the formation of hydrogen bonds. Meanwhile, APTES-modified mesoporous silica nanoparticle (AMSN)/TGF-β1 siRNA complexes were incorporated to facilitate the cellular uptake of siRNA and enable their escape from lysosomes. The localized co-delivery of 5-FU and TGF-β1 siRNA induces residual tumor cells killing by silencing TGF-β1 expression and reverses MMT. The combination of FC@MTs was shown to have a synergistic anti-peritoneal metastasis and anti-adhesion effects, which could be an effective strategy to enhance the clinical therapeutics of CRC.
Cancer-associated fibroblasts (CAFs) are the primary source of collagen I, which contributes to the formation of a dense tumor extracellular matrix (ECM). Non-selective targeting of collagen I through CAF inhibition may inadvertently promote tumor cell detachment and metastasis by triggering anoikis resistance. To address this, a "wandering tumor cells" strategy is proposed, combining the induction of tumor cell deadhesion with the reversal of anoikis resistance. For heterologous targeted drug delivery, thermosensitive lipids and a photosensitizer are incorporated into M1-type macrophage membranes (TMs) to enable laser-responsive activation. Based on this approach, we designed a photothermally triggered, functional macrophage membrane-camouflaged nano-cracker (TM@cP/siF-ErN) with a particle size of 162.20 ± 0.54 nm for the co-delivery of erianin (Er) and focal adhesion kinase small interfering RNA (siFAK). Er is encapsulated in anisamide (AA)-modified nanodiscs (ErN) with hydrodynamic diameter of 15.07 ± 7.24 nm to selectively inhibit collagen I synthesis in CAFs by targeting pyruvate carboxylase, thereby inducing tumor cell deadhesion. siFAK is delivered to tumor cells using cinnamaldehyde-modified polyethyleneimine (cP/siF) to formed complexes with a particle size of 98.57 ± 1.47 nm and enhance transfection efficiency, enabling effective FAK knockdown and reversal of tumor anoikis resistance. Furthermore, TMs are fragmented into debris to amplify M2-type macrophage repolarization. Experimental results show that the nano-cracker efficiently targets orthotopic 4T1 breast tumors and, upon laser-triggered detonation, releases ErN, cP/siF and M1-type macrophage membrane fragments, which collectively promote tumor anoikis by suppressing collagen I synthesis in CAFs and reversing tumor cell anoikis resistance. Moreover, it promotes the repolarization of M2-type macrophages, which synergizes with collagen I downregulation-induced infiltration of CD8⁺ T lymphocytes to enhance the antitumor immune response, collectively resulting in pronounced breast cancer suppression. This nano-cracker implements the "wandering tumor cells" strategy, offering a promising approach for improving tumor therapy and enabling heterologous targeted delivery.
Antisense oligonucleotides offer a powerful strategy for suppressing pro-inflammatory microRNAs, but efficient long-term delivery after systemic administration remains challenging. In this study, we developed a self-assembling oligoDNA-nanomicelle (OD-micelle) platform to simultaneously deliver antisense oligoDNA targeting miR-155 and curcumin, a hydrophobic anti-inflammatory agent, to the lung. The curcumin formulation (OD-micelle/Cur) can be administered intravenously and has a negatively charged surface and average particle size of ∼160 nm, supporting scavenger receptor (SR)-mediated pulmonary delivery. Fluorescence imaging and flow cytometry analyses demonstrated that cellular uptake was comparable to that of OD-micelle/PEI25k, a widely used cationic carrier standard. Hemocompatibility tests demonstrated reduced red blood cell aggregation, compared with PEI25k, indicating improved hemocompatibility without compromising delivery efficiency. Mechanistic studies supported the receptor-dependent transport of the oligoDNA corona. Pre-treatment with excess oligonucleotides reduced the cellular uptake and in vivo lung accumulation of Cy5-labeled OD-micelle/Cur. Furthermore, a RAGE antagonist peptide similarly reduced cellular uptake, suggesting the involvement of RAGE in the SR pathway. In LPS-induced acute lung injury (ALI) mice and LPS-stimulated Raw264.7 cells, the OD-micelle/Cur suppressed the inflammatory response, decreased TNF-α and IL-6 levels, and improved lung histopathology. The antisense oligoDNA corona contributed to the efficacy through miR-155 inhibition, which was confirmed by comparison with scrambled OD-micelle/Cur and increased SOCS1 expression in lung tissue. Furthermore, RAGE pathway inhibition attenuated the inflammatory response, suggesting that RAGE signaling could be an additional therapeutic mechanism. Therefore, OD-micelles are a systemically administrable, lung-targeted oligonucleotide nanoplatform with dual-mechanism anti-inflammatory activity for the treatment of ALI.
The Biopharmaceutics Classification System(BCS)serves as a foundational framework for drug development and,streamlines generic drugs approval by categorizing them based on solubility and intestinal permeability[1].
Osteoporosis is a skeletal disorder characterized by an imbalance between bone formation and resorption, which leads to progressive bone loss and increased fracture risk. While current treatments either inhibit bone resorption or stimulate bone formation, their long-term use is associated with adverse effects, necessitating alternative therapeutic approaches. In this study, we explore the use of red ginseng-derived nanovesicles (RGNVs) as a biocompatible nanotherapeutic strategy for treating osteoporosis. The RGNVs were successfully isolated and characterized, revealing a lipid bilayer structure enriched in bioactive ginsenosides and functional proteins. In vitro, RGNVs enhanced osteoblast proliferation, differentiation, and mineralization while suppressing osteoclast differentiation and bone resorption by modulating the BMP-2/Smad and MAPK signaling pathways. In an ovariectomy-induced osteoporosis mouse model, oral administration of RGNVs significantly restored bone volume and mineral density, and biodistribution studies confirmed their preferential accumulation in the bone tissue. Systemic toxicity evaluation indicated no adverse effects, supporting the safety of RGNVs for therapeutic use. These findings suggest that RGNVs regulate bone remodeling through a dual mechanism, to stimulate bone formation and inhibit bone resorption, thereby offering a promising and well-tolerated approach for osteoporosis management.
Endoplasmic reticulum stress (ERS), arising from the disruption of proteostasis within the tumor microenvironment, represents a fundamental driver of tumorigenesis, immune evasion and resistance against conventional therapies. In recent years, the precise modulation of ERS through the application of nanotechnology has emerged as a promising strategy to enhance the efficacy of cancer immunotherapy. This review provides a comprehensive analysis of the molecular mechanisms underlying ERS and discusses how engineered nanotherapeutics can selectively target the endoplasmic reticulum through approaches such as ligand conjugation, peptide modification or membrane fusion to induce sustained ERS. These nanotherapeutics initiate ERS by mechanisms including calcium ion dysregulation, overproduction of reactive oxygen species and direct activation of unfolded protein response signaling pathways. Persistent ERS subsequently facilitates immunogenic cell death by promoting the release of damage-associated molecular patterns, which enhance the maturation of dendritic cell and promote the activation of cytotoxic T lymphocytes. Moreover, combining ER-targeted nanotherapeutics with established therapeutic modalities, such as photodynamic therapy, photothermal therapy and chemodynamic therapy, has demonstrated synergistic antitumor efficacy and improved immune responses. Despite these advances, several critical challenges remain, particularly in terms of delivery efficiency, targeting specificity and systemic biocompatibility. Future research should emphasize the integration of nanotechnology with systems immunology and cancer metabolism, as well as the incorporation of artificial intelligence and single-cell omics to optimize the design and translational potential of ER-targeted nanotherapeutics. Collectively, these interdisciplinary strategies offer considerable potential to overcome therapeutic resistance and to promote the advancement of precision oncology.
Traditional Chinese medicine (TCM) has abundant medicinal resources and distinctive pharmacological properties. So, TCM presents considerable promise in clinical treatments. However, challenges such as poor bioavailability hinder broader clinical adoption of TCM. Microneedles (MNs), an innovative and minimally invasive transdermal platform, have emerged to enhance the therapeutic performance of TCM. The integration of MNs with TCM (TCM-MNs) overcomes key limitations of conventional administration routes to reach more targeted and efficient delivery. The structural and compositional diversity of TCM ingredients necessitates diverse TCM-MNs designs, especially "unification of medicines and excipients". Moreover, TCM-MNs can achieve synergistic benefits when combined with modality-specific interventions, like acupuncture. This review outlines advantages and types of TCM-MNs, according to the special structure and function of TCM components. Current applications of TCM-MNs in different diseases are also discussed. The review offers a promising foundation for the advancement and clinical translation of TCM-MNs in transdermal therapy.
Glioblastoma (GBM) is shielded by both the blood-brain barrier (BBB) and an immunosuppressive tumor microenvironment. Here, we develop a chimeric biohybrid nanovesicle (BEV-RVG29-PTX) that integrates viral tropism, bacterial vesiculation, and chemotherapeutic cytotoxicity into a single genetically programmable platform. Genetic fusion of rabies virus glycoprotein 29 (RVG29) to the AIDA1 autotransporter translocator domain enables robust, autonomous surface expression on bacterial extracellular vesicles (BEVs) without the need for chemical conjugation. The BEV-RVG29-PTX drives receptor-dependent BBB transcytosis and achieves efficient glioma accumulation. Encapsulated paclitaxel (PTX), otherwise restricted by BBB impermeability, is effectively delivered to intracranial tumors and induces reactive oxygen species-driven immunogenic cell death. Bone marrow-derived dendritic cells immune-activation experiments further confirmed an approximately 2-fold increase in CD80/CD86 activation. Synergizing with the pathogen-mimetic characteristics of BEVs, these signals also elicit an approximately 2-fold increase in intratumoral CD8⁺ T-cell infiltration, overcome immune exclusion, and achieve durable tumor control with extended survival in orthotopic GBM models. Accordingly, this virus-bacteria-drug biohybrid strategy enables targeted brain delivery while simultaneously amplifying antitumor immunity, offering a promising and translatable approach for GBM treatment.
The complicated and immunosuppressive tumor microenvironment usually obstruct the efficiencies of various therapeutic schedules including immunotherapy. Here, we report a programmable polymer-based nanoreactor for photothermal-enhanced immunotherapy through second near-infrared (NIR-II) light-triggered enzyme‑catalyzed immunogenic tumor microenvironment remodelling. The nanoreactor system contains a thermal-responsive liposome modified on its surface with xanthine oxidase (XO), and a core co-loaded with a NIR-II-absorbing semiconducting polymer, an oxygen carrier perfluorohexane (PFH) and a hypoxanthine substrate. Under NIR-II laser irradiation, the semiconducting polymer (SP-II) generates a local photothermal effect, directly ablating tumor cells and triggering a phase transition of the liposome shells, enabling precise pulsed release of the loaded contents. The released PFH rapidly alleviates local tumor hypoxia, providing a key substrate for subsequent enzyme cascade reactions. Simultaneously, hypoxanthine is catalyzed by XO to continuously generate superoxide anions and uric acid. In this approach, superoxide anions acting as reactive oxygen species enhance immunogenic cell death and oxidative stress, while uric acid serves as an endogenous danger signal, promoting M2 to M1 repolarization of tumor-associated macrophages, thereby synergistically remodeling the immunosuppressive tumor microenvironment. This strategy potently inhibits laser-irradiated primary tumors, as well as significantly suppresses the progress of distant and metastatic tumors, and prolongs the survival of mouse. Our study provides a new approach for developing programmable anti-tumor nanoreactors with enzyme-catalyzed immunogenic tumor microenvironment remodelling capabilities.
Lysosomal storage diseases (LSDs) are a group of inherited metabolic disorders caused by misfolding of lysosomal proteins and their degradation via endoplasmic reticulum-associated degradation (ERAD). Deficiency in LSD-associated enzymes leads to the accumulation of toxic materials within the lysosome. In macroautophagy (hereafter autophagy), autophagic receptors as represented by p62/SQSTM1/Sequestosome-1 collect and deliver their cargoes to the lysosome. Here, we developed the LYSOTAC (LYSOsome-TArgeting Chimera) technology, which enables lysosomal targeting of LSD-associated enzymes while preserving their enzymatic activities. LYSOTAC employs a bifunctional chimera that simultaneously binds an LSD-associated enzyme via the enzyme-binding ligand (EBL) and p62 via the autophagy-targeting ligand (ATL). Upon binding, p62 undergoes self-polymerization to form cargo-p62 complexes, which are sequestered into autophagosomes and delivered to lysosomes, where the enzymes exhibit maximal activity. Here, LYSOTAC compounds targeting β-glucocerebrosidase (GCase) were designed to restore GCase activity in lysosomes and promote glucosylceramide degradation in Gaucher disease fibroblasts. We suggest that LYSOTAC provides a potential therapeutic strategy for LSDs.
