Papers, explained in our own words

1. Lipoplex Nanoparticle-Based RNA Delivery to 3D Melanoma Spheroids

Rudolf R, Cesetti T, Keller F, Hafner M

Lipoplex nanoparticle-based vaccination technologies have shown tremendous efficiency for in vivo transfection during the COVID-19 pandemic and also hold promise for treating melanoma. While pharmacodynamics and pharmacokinetics need careful in vivo and clinical testing, other parameters of drug performance can be…

• CCD-1137Sk
• Fibroblast
• HaCaT
• Keratinocyte
• LPX
• Melanoma cell
• SK-MEL-28
• Spheroid
• Transfection

2. When structural sophistication becomes a translational barrier: the complexity trap in DNA nanocarrier design

Tunc Cu, Saha Sk, Panda Ss, Mori S, Culha M

DNA nanotechnology has emerged as a highly programmable platform in nanomedicine, enabling the fabrication of precise nanoscale architectures for drug delivery, biosensing, gene regulation, immunotherapy, and theranostics. DNA-based nanocarriers offer distinctive advantages, including structural programmability…

• DNA nanotechnology
• barrier
• drug delivery
• nanocarrier
• nanomedicine
• translation

3. Rivastigmine and Emerging Nanotechnological Strategies for Targeted Delivery: A Comprehensive Review

Babar E, Ur Rehman A, Gul P, Arshad Ms, Shah Ba, Ahmed N

Alzheimer's disease affects millions of people globally; it is an irreversible and incurable disease, characterized by gradual cognitive decline as well as pathophysiological changes including the presence of senile plaques and neurofibrillary tangles. There are several risk factors associated with Alzheimer's…

• Alzheimer’s
• Rivastigmine
• blood-brain barrier
• nanomedicine
• nanotechnology
• targeted delivery

4. Poly(acrylamido) PEG-Alternative Lipids Rescue mRNA Efficacy during Repeated Dosing and Enhance Immune Cell Delivery

Fiedler Bm, Galley C, Strimaite M, Cheng Nm, Mahmoudi N, Feng Z, Et Al.

Following the successes of the messenger RNA (mRNA) lipid nanoparticle (LNP) vaccines against COVID-19, mRNA-LNPs are being explored for varied multidose applications including cancer immunotherapies and protein replacement therapies. LNPs rely on poly(ethylene glycol)-lipid (PEG-lipid) coatings for stability, but…

• PEG alternative
• accelerated blood clearance
• anti-PEG antibody
• dendritic cell
• lipid nanoparticle
• mRNA delivery
• poly(acrylamido) lipid

5. Renal CLCN5 gene therapy for Dent disease type 1: local rapamycin immune modulation and age-dependent outcomes with lentiviral and AAV9 vectors

Lyu P, Bostanci A, Jiang C, Yoo Kw, Broadhurst R, Atala A, Et Al.

Dent disease type 1 is a rare X-linked renal tubulopathy caused by loss of the Cl⁻/H⁺ exchanger ClC-5, leading to defective proximal tubular endocytosis and low-molecular-weight proteinuria. Viral gene therapy represents a promising disease-modifying strategy; however, the relative performance of integrating versus…

6. CRISPR/Cas9-based Genome Editing in Inherited Diseases: Mechanisms, Therapeutic Advances, Safety and Clinical Translation

Fatima J, Siddique Yh

Gene therapy has transformed modern biomedical research by enabling curative strategies that target the molecular basis of disease. Among genome-editing technologies, the CRISPR/Cas9 system has emerged as a versatile and efficient platform due to its programmability, simplicity, and precision. This review summarizes…

• CRISPR/Cas9
• base editing
• inherited diseases
• non-homologous end joining
• prime editing

7. Doxorubicin Nanocarriers in Glioblastoma Therapy: Advances and Limitations

Colpan Rd

Glioblastoma (GBM) remains one of the most aggressive brain malignancies, with limited therapeutic success largely due to the restrictive nature of the Blood-Brain Barrier (BBB) and intrinsic drug resistance mechanisms. Despite standard treatment strategies, the median survival remains approximately 16 months…

• Doxorubicin
• blood-brain barrier
• brain cancer
• drug delivery systems
• glioblastoma
• nanocarriers

8. Reconstitution of lipid nanoparticle-mediated siRNA transfer across endosomal membrane mimics

Spadea A, Singh A, Redlingshoefer L, Sorrentino S, Kalaidzidis Y, Zerial M

Endosomal escape limits RNA delivery by lipid nanoparticles (LNPs). If escape requires membrane rupture, productive delivery would be intrinsically coupled to membrane damage and its potential toxicity. Here, we establish a cell-free system based on giant unilamellar vesicles (GUVs) with an early endosome-like lipid…

9. Circular RNA in Lipid Nanoparticles: From Molecular Engineering to First-in-Human Testing

Zhao Y, Cheng H, Zhou S, He L, Xiu C, Huang B, Et Al.

Circular RNAs (circRNAs) were, until a decade ago, a by-product of transcriptome profiling. They are now drug candidates. A covalently closed backbone removes the termini that exonucleases and cap-dependent decay require, and lipid nanoparticles (LNPs) turn that molecular property into a pharmacological one by…

10. Selection of Optimal AAV Serotype for Brain-Wide Gene Delivery Following Intracerebroventricular Injection in mdx52 Mice

Nagy C, Depond C, Doisy M, Vacca O, Goyenvalle A

Adeno-associated virus (AAV)-based gene therapy is a promising approach for the treatment of Duchenne muscular dystrophy. In addition to muscle degeneration, patients exhibit cognitive and behavioral impairments, highlighting the need for efficient brain targeting. While several AAV serotypes efficiently transduce…

11. Microfluidic lipid nanoparticles co-delivering siMOB1A and paclitaxel for ovarian cancer therapy

Xu Jy, Liu K, Chen Ly, Liu C, Chen Dy, Wu Sg, Et Al.

Ovarian cancer is the foremost cause of mortality among gynecologic malignancies. Paclitaxel (PTX) exerts a significant therapeutic effect on ovarian cancer by targeting microtubules and impeding the cell cycle progression during the mitosis phase. MOB kinase activator 1 A (MOB1A) has been proven to regulate the…

• Lipid nanoparticles
• Microfluidic technology
• Ovarian cancer
• Paclitaxel
• siMOB1A

12. Combined siRNA Targeting IκBα and Stat6 Promotes Macrophage Reprogramming toward Cancer Immunotherapy

Gul S, Vergnaud J, Wang Q, Hery M, Dumont F, Tran P, Et Al.

Tumor-associated macrophages (TAMs) are central regulators of the tumor microenvironment (TME). Their polarization state can critically influence tumor development and treatment resistance. In the TME, the pro-tumor M2-like phenotype is the most prevalent among TAMs and contributes to tumor progression by promoting…

• Csf-1r
• IκBα
• Stat6
• lipid nanoparticles
• macrophage reprogramming
• siRNA

13. A single liver-sparing myotropic AAV ameliorates muscular dystrophy in humanized mice by base editing

Pien Yc, Li H, Bulliard G, Liu H, Durbacz Mz, Xu Z, Et Al.

Duchenne muscular dystrophy (DMD) is caused by mutations in the DMD gene, most of which are large exon deletions that result in loss of dystrophin protein. Adenine base editing (ABE) enables permanent gene correction by modifying splice sites to cause exon skipping and restoration of the DMD open reading frame. We…

• Genetics
• Muscle biology
• Skeletal muscle

14. Efinaconazole-Loaded Liposomal Nanocarriers: A Box-Behnken Design Approach in Formulation Designing and Optimization for Onychomycosis

Singh M, Soni Pk, Soni R

To design, optimize, and evaluate efinaconazole-loaded liposomal formulation with sustained drug release, enhanced trans-ungual drug penetration, and improved drug retention for treatment of onychomycosis. Conventional topical therapies for onychomycosis are limited by poor nail permeability and low drug retention…

• Box-Behnken design
• Efinaconazole
• liposomes
• onychomycosis
• optimization
• transungual

15. Preparation and comprehensive physicochemical characterization of trastuzumab-loaded PEGylated liposomal nanoparticles for systemic delivery

Abbas Ss, Hassan Bar, Hadi Ha, Mohammed Ah, Alsammarraie Aza, Al-Bayati Ma

ObjectiveThe primary objective of this experimental formulation-development study was to prepare and physicochemically characterize a trastuzumab-loaded liposomal formulation containing post-inserted DSPE-PEG2000 for systemic-delivery research. The secondary objective was to determine whether the preparation and…

• HER2
• PEGylated liposomes
• monoclonal antibody delivery
• physicochemical characterization
• protein integrity
• systemic nanomedicine
• trastuzumab

16. mRNA Delivery with Lipopolymers to Rejuvenate TRAIL Therapy for Lung Cancer Treatment

Kc R, Nasrullah M, Meenakshi Sundaram Dn, Patel D, Sandhu G, Kucharski C, Et Al.

Tumor Necrosis Factor-Related Apoptosis-Inducing Ligand (TRAIL) has long been identified as a promising therapeutic due to its ability to induce apoptosis selectively in cancer cells. However, poor stability and rapid clearance of the administered TRAIL protein in circulation have hampered its clinical development…

• TRAIL
• gene therapy
• lung cancer
• mRNA
• transfection

17. Rapamycin-loaded chitosan functionalized poly (lactic-co-glycolic acid) (PLGA) nanoparticles for potential wet age-related macular degeneration treatment: an in vivo study

Hu Y, Wei L, Yang H, Sun J, Li D, Liu Q

Wet age-related macular degeneration (Wet AMD) is a major cause of vision loss and has been linked to abnormal choroidal neovascularization (CNV). Current therapies require frequent intravitreal injections, which leads to poor patient adherence. Herein, this study was designed to investigate rapamycin-loaded…

18. Effect of Glucose and Methyl Glucoside on the Structure and Physicochemical Properties of Liposomes

Sekine A, Tsuchiya K, Ogura T, Akamatsu M, Arakawa K, Sakai K, Et Al.

Sugars have been widely used as humectants and cryoprotectants in the cosmetics and pharmaceutical industries; thus, investigating their effects on the structure and properties of lipid bilayers can provide valuable insights for the development of functional lipid nanoparticles and drug delivery systems. In this…

• Raman spectroscopy
• freeze-fracture transmission electron microscopy
• glucose
• liposome
• small angle x-ray scattering

19. Reln mRNA Delivery Drives Neurogenic Bone Regeneration by Orchestrating Schwann Cell-Mediated Exosomal Trafficking

Lin S, Li Q, Liu D, Li M, Zhang H, Chen L, Et Al.

Neurogenic bone regeneration is essential for the effective restoration of bone tissue functionality, while the mechanisms and new strategies underlying neurogenic bone regeneration remain to be further explored. Here, single-cell transcriptomic analysis revealed enhanced mesenchymal-Schwann cell crosstalk during…

• Reelin
• Schwann cells
• exosomal endocytosis
• mRNA delivery
• neurogenic bone regeneration

20. Protocol for comprehensive genotyping of nuclease, base, and prime editing outcomes using CleanFinder

Dobner J, Ramachandran H, Rossi A

Genome editing experiments often produce heterogeneous allele mixtures that require structured genotyping. Here, we present a protocol for analyzing amplicon sequencing data from nuclease, base, prime, and mitochondrial base editing experiments using CleanFinder. We describe steps for preparing reference sequences…

• Bioinformatics
• CRISPR
• Genomics

21. BBR-RHE Co-Loaded Nanoemulsion Potentiates Anti-PD-L1 Efficacy via Lipid Metabolic Reprogramming in Triple-Negative Breast Cancer

Wei J, Qian K, Shi J, Chen K, Zhang H, Gu M, Et Al.

Triple-negative breast cancer (TNBC) therapy is limited by the poor efficacy of anti-programmed death-ligand 1 (aPD-L1) monotherapy, with dysregulated lipid metabolism in the tumor microenvironment (TME) driving immunosuppression. Herein, a nanoemulsion co-loaded with berberine (BBR) and rhein (RHE) (BBR-RHE-NE), two…

• aPD-L1
• berberine
• lipid metabolism
• rhein
• triple-negative breast cancer

22. Protein adsorption and drug delivery potential of graphitic carbon nitride nanorods: physicochemical insights with human serum albumin and quercetin

Pasangha Ch, Kishore N

The two-dimensional polymeric material graphitic carbon nitride (gCN) has remarkable stability, with applications in photothermal therapy and drug delivery. When nanomaterials encounter proteins, they can lead to protein adsorption on their surfaces or the development of a protein corona. Such interactions could…

23. Novel modifications of small extracellular vesicles to enhance cardiac-specific delivery of siPKA RNA and edaravone against ischemia-reperfusion cardiac injury

Pei X, Li R, Bai Y, Yan Q, Qi X, Jiao W, Et Al.

Relative tight endothelium in the heart may be a barrier for drugs in the large molecule and nanoparticle formats to act on cardiomyocytes. We hypothesize that enhanced binding of large molecules or nanoparticles to the cardiac capillary endothelial cells can increase the transendothelial transport of these drugs to…

• Cardiac-specific transendothelial transport
• Edaravone
• Multistage-targeting drug delivery system
• Myocardial ischemia-reperfusion injury
• PKA siRNA
• Permanent MI

24. A multimodal anticancer strategy based on exosome-coated prussian blue nanocarriers for melanoma treatment

Roshani R, Ramezani M, Bagheri E, Alikhani M, Abnous K, Taghdisi Sm, Et Al.

Overcoming multidrug resistance and tumor recurrence in melanoma requires multimodal therapeutic strategies that surpass the limitations of monotherapy. Herein, we report a biomimetic nanoplatform, HMPB@EX-DOX, designed to combine chemotherapy, photothermal therapy (PTT), and iron-mediated oxidative stress to…

• Exosome
• Melanoma
• Oxidative stress
• Photothermal therapy
• Prussian blue nanocubes