bims-supasi Biomed News
on Sulfation pathways and signalling
Issue of 2025–05–18
eighteen papers selected by
Jonathan Wolf Mueller, University of Birmingham



  1. Chem Commun (Camb). 2025 May 15.
      Herein, we report the synthesis of heparan sulfate (HS) proteoglycan mimetics bearing iduronic acid (IdoA) and sulfated L-idose (Ido) hexasaccharides to assess how these isostructural sugars with similar charge density influence neoproteoglycan display on the cell membrane. PG@I2, carrying sulfated L-idose, showed rapid internalization in both cancerous and normal cells, whereas PG@I1, containing native IdoA expressed on the cell membrane and slowly internalized, underscoring the role of IdoA in HSPG cell surface engineering.
    DOI:  https://doi.org/10.1039/d5cc00527b
  2. J Agric Food Chem. 2025 May 14.
      Heparin, as a type of highly sulfated polysaccharide, is crucial for various physiological and pathophysiological functions. Heparan sulfate 2-O-sulfotransferase (2OST) is responsible for the second sequential sulfation modification in heparin biosynthesis. However, challenges such as low expression and poor enzyme activity performance limit its application. To this end, a combination of strategies was employed to improve expression level and catalytic performance. First, SUMO fusion tag was used to enable the active expression of Gallus gallus-derived 2OST (Ga2OST), followed by enhancing its expression level through N-terminal synonymous codon optimization. Under the principle of considering both catalytic activity and stability, the combinatorial mutant SUMO-Ga2OST A98K/Y145F was successfully constructed, resulting in a 2.32-fold increase in catalytic activity and a 7.80-fold extension of its half-life. Eventually, the enzyme activity was improved to 5720 U/mL with a 14.79-fold increase in a 5 L fermenter, which, to the best of our knowledge, is the highest reported to date. The engineered mutant SUMO-Ga2OST A98K/Y145F with markedly enhanced active expression and catalytic performance could provide a solid foundation for heparin biomanufacturing.
    Keywords:  catalytic performance; enzyme engineering; expression level; heparan sulfate 2-O-sulfotransferase; heparin; sulfation
    DOI:  https://doi.org/10.1021/acs.jafc.5c00771
  3. Int J Biol Macromol. 2025 May 09. pii: S0141-8130(25)04635-5. [Epub ahead of print]311(Pt 4): 144083
      Targeted drug delivery strategy can accurately deliver multiple drugs to tumor cells by biocompatible materials, enhance the therapeutic effect and weaken adverse side effects. Chondroitin sulfate (CS) is a biodegradable material which has dual-targeting ability to CD44 receptors and Golgi apparatus. Flurbiprofen is a COX-2 inhibitor that can be used as an anticancer adjuvant in combination with docetaxel to enhance the antitumor effect. Herein, docetaxel and flurbiprofen were coupled to CS via disulfide bond and ester bond respectively to prepare polymer conjugates chondroitin sulfate-docetaxel (C-ss-D) and chondroitin sulfate-flurbiprofen (C-F). Then the reduction-sensitive micelles C-ss-D and the pH-sensitive micelles C-F were constructed by self-assembly respectively, and the dual-drug micelles C-F/C-ss-D were further prepared. C-F/C-ss-D displayed uniform spherical shape, negative surface charge, and achieved controlled drug release in slightly acidic and reductive tumor microenvironment. C-F/C-ss-D exhibited remarkable targeting ability towards tumor cells and Golgi apparatus, demonstrated potent cytotoxic effect on MCF-7 cells, and induced apoptosis by regulating the expression of COX-2 and apoptosis-related proteins. C-F/C-ss-D effectively inhibited tumor growth in MCF-7 xenograft mice with low toxicity to blood and major organs. Therefore, the environmental responsive dual-drug synergistic and dual-targeted micelles based on CS have great potential for the treatment of breast cancer.
    Keywords:  Chondroitin sulfate; Dual-targeting micelles; Environmental response
    DOI:  https://doi.org/10.1016/j.ijbiomac.2025.144083
  4. ACS Chem Biol. 2025 May 13.
      Identifying smaller sulfated glycan fragments that recognize target proteins with high affinity is highly challenging. In this work, we show that microarray screening of 53 small glycan fragments helped identify distinct sulfated monosaccharide to tetrasaccharide fragments that bind to multiple isoforms of SARS-CoV-2 spike glycoprotein (SgP) with high affinity. Our library consisted of natural and unnatural glycan sequences with a wide range of sulfation levels. The unnatural features arose from the presence of phosphate or fluoro groups on the natural sulfated GAG scaffold as well as sulfate modification of idose fragments that were monomer to tetramer long. None of the natural glycans yielded much promise, which probably conveys the importance of the polymeric glycosaminoglycan chain in SgP biology. However, the unnatural idose fragments with sulfation at the 2, 3, 4, and 6 positions displayed high affinities (100-500 nM) for wild-type, Delta, and Omicron variants of SgP. The unnatural sulfated idose monosaccharide is the smallest molecule known to date that can be classified as a high-affinity, pan-variant fragment. This fragment is expected to serve as the lead for the design of pan-variant ligands with sub-nM inhibition potency.
    DOI:  https://doi.org/10.1021/acschembio.5c00206
  5. Int J Mol Sci. 2025 May 03. pii: 4350. [Epub ahead of print]26(9):
      Defective CFTR (cystic fibrosis transmembrane conductance regulator) is pathognomonic for cystic fibrosis (CF), which is characterized by an accumulation of tenacious secretions in pulmonary airways, as well as by abnormal ductal secretions in other organs, including the pancreas and prostate. The advent of CFTR modulating therapies has markedly improved the clinical status and survival of CF patients, primarily attributable to improved lung function. Previous publications reported that a decline in CFTR function was associated with a decline in activity and expression of the enzyme N-acetylgalactosamine-4-sulfatase (Arylsulfatase B; ARSB). ARSB removes 4-sulfate groups from N-acetylgalactosamine 4-sulfate residues and is required for the degradation of chondroitin 4-sulfate (chondroitin sulfate A) and dermatan sulfate, two sulfated glycosaminoglycans which accumulate in cystic fibrosis. Declines in both ARSB and in CFTR have been associated with the development of malignancies, including prostate malignancy. The experiments in this report show that similar effects on invasiveness are present when either CFTR or ARSB is inhibited in human prostate epithelial cells, and these effects resemble findings detected in malignant prostate tissue. The effects of CFTR inhibition are reversed by treatment with recombinant human ARSB in prostate cells. These results suggest that treatment by rhARSB may benefit patients with cystic fibrosis and prostate cancer.
    Keywords:  Arylsulfatase B; CFTR; chondroitin sulfate; cystic fibrosis; glycosaminoglycans; prostate cancer
    DOI:  https://doi.org/10.3390/ijms26094350
  6. Int J Biol Macromol. 2025 May 10. pii: S0141-8130(25)04669-0. [Epub ahead of print] 144117
      Sulfated polysaccharides from edible marine algae have gained significant attention for their remarkable nutritional and therapeutic potential. Caulerpa racemosa (sea grape), a widely consumed edible algae, is a source of bioactive compounds with significant medicinal potential. This study focuses on CRP-2, a sulfated (1 → 4) galactan derived from C. racemosa, and its anti-inflammatory effects in lipopolysaccharide (LPS)-induced CALU-1 cells. CRP-2 (at 125 μg/mL) reduced expressions of pro-inflammatory cytokines (interleukin-1β and tumor necrosis factor-α) by 41-49 % in CALU-1 cells, along with the downregulation of transforming growth factor-β (mean fluorescence intensity reduced from 20 to 4) level, in comparison with LPS treatment. Concurrently, downregulated expressions level of interleukin-33, interferon-α and interleukin-10 in LPS-induced CALU-1 cells (mean fluorescence intensities 8-11fold) were substantially elevated after the treatment with CRP-2 (mean fluorescence intensities 25-35-fold), in a dose-dependent manner. Gene expression analysis by qRT-PCR revealed downregulation of interferon-γ levels by 2 folds (at 125 μg/mL) from an overly expressed levels of 9-fold in LPS induced cells. Structure-activity and molecular docking analyses emphasized the role of sulfate moieties in its bioactivity. These findings highlight the potential of CRP-2 as a natural anti-inflammatory therapeutic and its health benefits as a functional food component.
    Keywords:  Cytokine regulation; Edible Sea grape Caulerpa racemosa; Sulfated (1 → 4) galactan
    DOI:  https://doi.org/10.1016/j.ijbiomac.2025.144117
  7. J Mater Chem B. 2025 May 14.
      Chronic, deep, and irregularly shaped wounds often infected with bacteria are considered a major clinical concern. The overproduction of reactive oxygen species (ROS) and disruption of the balance between pro-inflammatory and anti-inflammatory cytokines delay the healing process. Traditionally used dressings are unable to address these multiple issues. We present a multifunctional, self-adaptable, injectable hydrogel composed of gelatin (G) and chondroitin sulfate (CS) containing borate-crosslinked tannic acid (TA), enriched with in situ synthesized silver nanoparticles (AgNPs), which eliminates the necessity of any secondary dressing. The dynamically crosslinked hydrogel demonstrates efficient self-healing, adhesiveness, antioxidant properties, and potential antibacterial activity (E. coli and S. aureus). The injectable hydrogels also exhibit sustained release of TA and AgNPs. The in vitro cytotoxicity reveals the excellent cytocompatibility of the hydrogel with HDF-N fibroblast cells and red blood cells. In vivo studies confirm that the injectable hydrogel demonstrates self-adaptability in irregularly shaped wounds and accelerates the healing process in terms of healing percentage, fibroblast generation, neovascularization, and hair follicle development. Additionally, the in vivo application of the fabricated hydrogels does not produce any significant systemic toxicity. This study demonstrates that the dynamically crosslinked, multifunctional, injectable hydrogel is a promising candidate for treating irregular deep penetrating wounds.
    DOI:  https://doi.org/10.1039/d4tb02537g
  8. Nat Commun. 2025 May 10. 16(1): 4364
      Fucoidan, a sulfated glycan derived from brown algae, has garnered significant attention for its anticoagulant properties. However, the structural complexity and heterogeneity of naturally extracted fucoidan have hindered a comprehensive understanding of its structure-activity relationship, limiting the development of fucoidan-based anticoagulant drugs. To address this challenge, we synthesize a diverse library of 58 distinct fucoidans with multiple contiguous 1,2-cis glycosidic bonds, ranging from disaccharides to dodecasaccharides, using a highly efficient preactivation-based one-pot glycosylation strategy. This library includes compounds with various sulfation patterns (2,3-O-di-, 3,4-O-di-, and 2,3,4-O-tri-sulfation) encompassing nearly all possible fucoidan structures. In vitro anticoagulant assays demonstrate that both molecular size and degree of sulfation play crucial roles in anticoagulant potency. Notably, compounds 29, 30, 37, and 58 significantly prolong human plasma activated partial thromboplastin time (APTT), comparable to the effect of enoxaparin, without affecting prothrombin time (PT) or thrombin time (TT). This selective inhibition of the intrinsic coagulation pathway suggests a reduced risk of bleeding, highlighting the therapeutic potential of these fucoidans as safer anticoagulant agents.
    DOI:  https://doi.org/10.1038/s41467-025-59632-2
  9. Int J Mol Sci. 2025 Apr 24. pii: 4037. [Epub ahead of print]26(9):
      Alzheimer's disease and other neurodegenerative disorders are characterized by the accumulation of misfolded proteins, such as amyloid-beta, tau, and α-synuclein, which disrupt neuronal function and contribute to cognitive decline. Heparan sulfate proteoglycans, particularly syndecans, play a pivotal role in the seeding, aggregation, and spreading of toxic protein aggregates through endocytic pathways. Among these, syndecan-3 is particularly critical in regulating the internalization of misfolded proteins, facilitating their propagation in a prion-like manner. This review examines the mechanisms by which syndecans, especially SDC3, contribute to the seeding and spreading of pathological protein aggregates in neurodegenerative diseases. Understanding these endocytic pathways provides valuable insights into the potential of syndecans as biomarkers and therapeutic targets for early intervention in Alzheimer's disease and other related neurodegenerative disorders.
    Keywords:  endocytosis; heparan sulfate proteoglycans; neurodegeneration; protein aggregation; syndecans
    DOI:  https://doi.org/10.3390/ijms26094037
  10. Sci Rep. 2025 May 14. 15(1): 16679
      Lichen planus (LP), an inflammatory and chronic disorder, is immune-mediated and influences nails, skin, hair, and mucosal tissues. Autoimmune diseases affect females more commonly, and it is reported that there is an association between certain sex hormones and autoimmune diseases. This study aims to evaluate the serum levels of DHT, Dehydroepiandrosterone (DHEAs), and testosterone (T) in patients who have oral lichen planus (OLP) compared to healthy people. Forty female patients diagnosed with OLP were selected. They were considered to have the same mean age as 40 patients in the control group. The enzyme-linked immunosorbent assay investigated Androgens' serum levels (Monobomal kit, Sherkat Azmayeshgah Bartar). The age mean 51.90 ± 11.52 for the patient group and 49.10 ± 11.63 for the control group. Erosive LP was the most common subtype of LP, 72.5% of LP lesions. The differences between DHEAs (p value = 0.126), DHT (p value = 0.710), and T's serum level (p value = 0.376) groups were not significant. There was no difference between hormonal levels during menopause and the non-menopause group. There was no correlation between OLP and androgens. DHEAs, DHT, and T levels were not different between case and control groups; therefore, the results did not support any neuroendocrine etiology for OLP. Also, there was no change in these androgens' serum levels during the transition from pre-menopause to post-menopause.Trial registration: The study adhered to the ethical principles of the Helsinki Declaration (2002 version) and received approval from the ethics committee of Shiraz University of Medical Sciences, Shiraz, Iran (IR.SUMS.REC.1397.709).
    Keywords:  Dehydroepiandrosterone; Gonadal steroid hormones; Lichen planus; Testosterone
    DOI:  https://doi.org/10.1038/s41598-025-01750-4
  11. Int J Biol Macromol. 2025 May 09. pii: S0141-8130(25)04638-0. [Epub ahead of print]311(Pt 4): 144086
      The rising global focus on sustainable algal biomaterials has catalyzed significant research into Ulva clathrata polysaccharides (UP), which are recognized for their renewable sourcing, biocompatibility, and multifaceted bioactivities. However, the limited mechanical strength of UP-based hydrogels has hindered their biomedical applications in drug delivery and wound healing. This study selected six extraction methods (hot water, acid, alkali, enzyme, ultrasound, and microwave) to obtain UP. It was found that there were differences in the molecular weight, sulfate group content, and monosaccharide composition of UP extracted from the same species by different methods. Subsequently, hydrogels were formed by cross-linking with trivalent cations, and it was observed that the mechanical properties of these six hydrogels differed significantly, with the main influencing factors being molecular weight, glucuronic acid, and sulfate group content. Among them, the hydrogels prepared from alkali-extracted polysaccharide (ALE) exhibited the best mechanical properties and a more homogeneous and denser microstructure. In addition, the ALE hydrogel maintained a stable state after swelling for 6 h. These findings provided novel insights for optimizing the weak gelling of U. clathrata polysaccharides and laid a theoretical foundation for developing and utilizing UP hydrogels.
    Keywords:  Extraction procedures; Hydrogel; Sulfated polysaccharide; Ulva clathrata
    DOI:  https://doi.org/10.1016/j.ijbiomac.2025.144086
  12. Biosens Bioelectron. 2025 May 08. pii: S0956-5663(25)00436-1. [Epub ahead of print]284 117562
      Sulfatase, traditionally known for its role in sulfate ester hydrolysis, has recently emerged as a potential player in tumor biology through its involvement in oxidative stress pathways. Here, we demonstrate for the first time that sulfatase exhibits peroxidase-like activity, catalyzing the generation of singlet oxygen (1O2) in the presence of oxygen. Based on the sulfatase-dependent 1O2 generation, the developed adamantly-enolether chemiluminescence probe QM-CF for imaging tumors of high sulfatase expression further verified the theory that sulfatase can be involved in tumor development. High-throughput screening (HTS) of natural compounds and clinical drugs identified scutellarin and sinomenine as potent sulfatase inhibitors that suppress tumor growth in mice. Mechanistic investigations revealed that these inhibitors modulate oxidative stress by downregulating MAPK and NF-κB pathways. Our findings unveil a previously unappreciated role of sulfatase in tumor-related oxidative stress and provide a promising platform for the discovery of novel sulfatase inhibitors, and advancing cancer therapeutics.
    Keywords:  (1)O(2); Chemiluminescence; High-throughput screening; Sulfatase
    DOI:  https://doi.org/10.1016/j.bios.2025.117562
  13. ACS Omega. 2025 May 06. 10(17): 18005-18016
      Arylsulfatase catalyzes the cleavage of sulfate ester bonds and plays a role in agar desulfation, thereby enhancing agar gel strength and quality. While studying the desulfurization pathway in Pseudoalteromonassp. SR43-6, a sequence encoding a potential arylsulfatase-Pseudoalteromonas Ars (Ps-Ars)-was found. The enzyme, with p-nitrophenyl sulfate as a substrate, exhibited optimal activity at 35 °C and pH 8.0. Its relative activity (206 U/mg) exceeded that of the recently identified arylsulfatases. Four hundred units of the enzyme removed 86.4% of sulfate groups from Gelidium amansii agar in 4 h, whereas 800 U of the enzyme removed 71.3% of sulfate groups from Gracilaria lemaneiformis agar in 8 h. After enzymatic treatment, G. amansii agar gel strength was enhanced by 32%, and a similar improvement was observed in G. lemaneiformis agar gel strength. Enzymatic agar desulfurization offers mild, quality-retaining, and environmentally friendly advantages, augmenting industrial application prospects.
    DOI:  https://doi.org/10.1021/acsomega.5c01356
  14. Int J Mol Sci. 2025 Apr 26. pii: 4120. [Epub ahead of print]26(9):
      Inflammation of the exocrine pancreas accompanies autoimmune diabetes in mouse models and humans. However, the relationship between inflammation in the exocrine and endocrine (islet) compartments has not been explored. To address this issue, we used a transgenic mouse model in which autoimmune diabetes is acutely induced after the transfer of islet beta cell-specific transgenic T cells. Histological analyses demonstrated that inflammation of the exocrine pancreas, which was initially mild, resulted in the transient but widespread disruption of acinar tissue. Islet inflammation preceded exacerbated exocrine pathology, progressed to T cell-induced islet damage/destruction and persisted when exocrine inflammation subsided. Heparanase-1 (HPSE-1), an endoglycosidase that degrades heparan sulfate in basement membranes (BMs), when preferentially expressed in recipient cells but not donor (HPSE-1-deficient (HPSE-KO)) T cells, played a critical role in both exocrine and islet inflammation. In this context, HPSE-1 facilitates the passage of autoimmune T cells across the sub-endothelial basement membrane (BM) of pancreatic blood vessels and initially into the exocrine tissue. Peak exocrine inflammation that preceded or accompanied the acute onset of diabetes and HPSE-1 potentially contributed to acinar damage. In contrast to inflammation, HPSE-1 expressed by donor T cells played a key role in the induction of diabetes by allowing autoimmune T cells to traverse peri-islet BMs in order to destroy insulin-producing beta cells. Overall, our findings suggest that major exocrine pancreas injury is not required for the initiation of autoimmune islet damage and is not essential at the time of diabetes onset.
    Keywords:  autoimmune diabetes; exocrine pancreas; heparanase-1 (HPSE-1); inflammation; islet
    DOI:  https://doi.org/10.3390/ijms26094120
  15. Mol Pharm. 2025 May 14.
      The introduction of chemical modifications to oligonucleotides has been pivotal to their emergence as effective therapeutic agents. Understanding how these modifications influence the higher-order structure of oligonucleotides is one of the key considerations during the drug development process. Here, we use a G-quadruplex (thrombin-binding aptamer) and stem/loop structure (dehydroepiandrosterone sulfate binding aptamer) as model cases to show for the first time that the stereochemistry of phosphorothioate linkages can affect the stability and function of oligonucleotides that depend on their intramolecular conformation for activity (e.g., aptamers and DNAzymes). Differences up to 15 °C in melting temperature are observed between stereochemical variants of the same sequence. Moreover, using a hemin-DNAzyme assay and small molecule recognizing fluorogenic aptamer, we illustrate that stereochemistry can be used to tune the binding affinity and activity of the oligonucleotides examined in this study toward non-nucleic acid targets. Taken together, these discoveries highlight the potential influence of stereochemistry on the structure and function of other phosphorothioate-containing oligonucleotides, revealing important considerations for future drug design.
    Keywords:  DNAzyme; formulation development; oligonucleotides; phosphorothioate; thrombin-binding aptamer
    DOI:  https://doi.org/10.1021/acs.molpharmaceut.5c00117
  16. Front Pharmacol. 2025 ;16 1577942
       Introduction: In this study, we investigated the protective effect of JBP485 against aristolochic acid I (AAI)-induced nephrotoxicity and explored the pharmacokinetic mechanisms. The effects of JBP485 on AAI-induced cytotoxicity and nephrotoxicity were evaluated in vitro and in vivo, respectively.
    Methods: To ascertain the protective effect of JBP485 against AAI-induced nephrotoxicity, we measured levels of urea nitrogen (BUN), creatinine (CRE), and indoxol sulfate in blood and urine; determined kidney weight-to-body weight ratio; and performed hematoxylin and eosin (H&E) staining. Cell viability and Western blotting assays, along with determination of malondialdehyde (MDA), superoxide dismutase (SOD), and intracellular reactive oxygen species (ROS) contents, were carried out to explore mechanisms underlying the protective effects of JBP485 against AAI-induced nephrotoxicity.
    Results: JBP485 treatment attenuated AAI-induced injuries in rat kidney while decreasing the levels of indoxyl sulfate, CRE, and BUN in plasma and increasing those of indoxyl sulfate in urine compared to that in AAI alone-treated group. The co-administration of JBP485 with AAI significantly increased the concentration and AUC of AAI in plasma, while decreasing its cumulative urinary excretion and renal clearance. Moreover, JBP485 reduced the uptake of AAI in kidney slices and human organic anion transporter 1/3 (hOAT1/3)-transfected human embryonic kidney 293 (HEK293) cells, suggesting that JBP485 ameliorated AAI-induced nephrotoxicity by reducing renal exposure to AAI via OAT inhibition. Meanwhile, JBP485 modulated the abnormal expressions of Oat1, Oat3, organic cation transporter 2 (Oct2), P-glycoprotein (P-gp), multidrug resistance-associated protein 2 (Mrp2) and multidrug and toxin extrusion proteins 1 (Mate 1) in rat kidney, suggesting that JBP485 improved tubular secretion in AAI-treated rats. Moreover, JBP485 reversed the AAI-induced changes in the expression of heme oxygenase 1 (HO-1), NAD(P) H: quinone oxidoreductase-1 (NQO1), B-cell lymphoma-2 (Bcl-2) protein expressions and Bcl-2-like protein 4 (Bax) induced by AAI in rat kidney. JBP485 increased cell viability and reduced intracellular levels of ROS in NRK-52E cells treated with AAI.
    Discussion: These results suggested that JBP485 protected against AAI-induced renal oxidative stress. All results indicated that JBP485 protected against AAI-induced nephrotoxicity by reducing renal exposure to AAI and alleviating oxidative stress. Our findings suggested that JBP485 has potential as a renoprotective agent for the prevention of AAI-induced nephrotoxicity.
    Keywords:  DDI; JBP485; OATs; aristolochic acid I; nephrotoxicity
    DOI:  https://doi.org/10.3389/fphar.2025.1577942
  17. Int J Pharm. 2025 May 10. pii: S0378-5173(25)00545-9. [Epub ahead of print] 125708
      Electroporation (EP) is a technique that temporarily increases cell membrane permeability through high-voltage electrical pulses, facilitating the internalization of hydrophilic drugs. When used in clinics, reversible EP offers significant advantages in drug delivery with minimal systemic toxicity, making it a promising approach in cancer therapy (Electrochemotherapy). However, is still challenging to increase therapeutic efficacy, such as increasing the amount of drug internalized by cells after EP. To address these limitations, integrating nanocarriers-particularly liposomes-into EP-based drug delivery strategies has shown great promise. Due to their structural similarity to cell membranes, liposomes can undergo electroporation without causing irreversible cell damage, enabling localized and controlled drug release at targeted sites. This study preliminary evaluates the effectiveness of positively charged gentamicin sulfate loaded liposomes (GS-Lipo) in enhancing gentamicin sulfate uptake through electroporation. The focus is on liposome behavior under EP, drug release, and cellular internalization. The results reveal a strong interplay between liposomes and EP. While EP minimally affects liposome size (sizes lower than 250 nm before and after EP) and PDI, it significantly enhances intracellular uptake and drug release by creating transient pores in liposomal bilayer, facilitating gentamicin diffusion. In vitro uptake studies performed with fluorescent liposomes and GS-Lipo, confirmed superior performance when combined treatment (EP + GS-Lipo) is used. By optimizing electroporation parameters (160 V, 200 V, and 250 V), this study succeeds in maximizing intracellular drug concentration, with the long-term goal of improving therapeutic outcomes, particularly in cancer treatment.
    Keywords:  Electroporation; Gentamicin Sulfate; Liposomes; Microfluidics
    DOI:  https://doi.org/10.1016/j.ijpharm.2025.125708
  18. J Vitreoretin Dis. 2025 May 10. 24741264251340108
      Purpose: To describe a patient presenting in adulthood with isolated retinopathy found to have mucopolysaccharidosis type IIIA. Methods: A single case was evaluated. Results: A 36-year-old man presented with 5 years of worsening peripheral vision and night vision. The initial examination and testing raised concern for rod-cone dystrophy. Genetic testing with an Invitae Inherited Retinal Disorders Panel showed 2 variants of SGSH, which is associated with mucopolysaccharidosis type IIIA. Laboratory testing showed low heparan-N-sulfatase levels and elevated heparan sulfate levels. These results and a thorough review of the literature support a diagnosis of mild attenuated non-neuronopathic mucopolysaccharidosis type IIIA. Conclusions: This case highlights the necessity for collaboration with genetic counselors and the value of a provider's clinical acumen in interpreting genetic testing results. Furthermore, the importance of considering mucopolysaccharidosis type IIIA when adult patients present with new-onset isolated retinitis pigmentosa is emphasized.
    Keywords:  Sanfilippo syndrome; lysosomal storage disorders; mucopolysaccharidoses; mucopolysaccharidosis type III; retinitis pigmentosa
    DOI:  https://doi.org/10.1177/24741264251340108