Papers, explained in our own words

97. 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

98. 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

99. Exosomes in regenerative medicine from biological messengers to engineered nanocarriers for precision therapeutics

Ghosh S, Bhaskar R, Singh Kk, Sharma S, Han Ss, Sinha Jk

Exosomes have emerged as a promising class of next-generation nanocarriers at the intersection of regenerative medicine and drug delivery. Compared with conventional synthetic nanocarriers and stem cell-based therapies, exosomes offer unique advantages, including intrinsic biocompatibility, low immunogenicity…

• Artificial Intelligence
• Exosomes
• Immunomodulation
• Nanocarrier
• Regenerative medicine

100. Biogenic gold nanoparticles in breast cancer nanomedicine: sustainable synthesis, multifunctional engineering, and the road to clinical translation

Singh Ur, Mishra G, Leharwani M, Abourehab Mas, Gupta G, Wen Goh K, Et Al.

The use of plant, algal, microbial or biomolecule based reducing and capping agents to synthesize gold nanoparticles (AuNPs) has paved the way for the use of these materials as versatile candidates for breast-cancer nanomedicine. In addition to being a more sustainable synthetic pathway, the biological elements…

• Biogenic gold nanoparticles
• Breast cancer
• Cancer nanomedicine
• Green synthesis
• Targeted drug delivery
• Theranostics

101. 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…

102. 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…

103. Translational Strategies in Smart Drug Delivery Systems for Controlled and Personalized Pharmacokinetics

Thalluri C, Bukke Spn, Medhi J, Vasam M, Vidiyala N, Sunkishala P, Et Al.

Advancements in drug delivery systems (DDSs) have developed pharmacotherapy by enabling precise control over drug pharmacokinetics (PKs), enhancing therapeutic efficacy, minimizing toxicity, and improving patient compliance. This review comprehensively examines emerging DDS strategies designed to optimize absorption…

• drug delivery systems
• nanotechnology
• personalized therapy
• pharmacokinetics
• stimuli-responsive DDS

104. 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…

105. Multifunctional Nanomedicine for Precision Theranostics in Hepatic Fibrosis: From Rational Design to Clinical Translation

Li Y, Gong Y, Li X, Yan S, Yu J, Qiu Z, Et Al.

Hepatic fibrosis (HF) is a pathological wound-healing response to chronic liver injury that can progress to cirrhosis and hepatocellular carcinoma. Although resmetirom and semaglutide have been approved for noncirrhotic metabolic dysfunction-associated steatohepatitis with fibrosis, they are restricted to a single…

• clinical translation
• hepatic fibrosis
• microenvironment-responsive targeting
• multifunctional nanomedicine
• precision theranostics

106. 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

107. Mathematical modeling in mRNA therapeutics: towards a pathway to individualized treatment protocols

Campanile E, Pettinà E, Marchetti L

mRNA-lipid nanoparticle (mRNA-LNP) therapeutics represent a transformative pharmacological paradigm in which the delivery vehicle, genetic payload, and expressed protein form a tightly coupled system. This multicomponent structure introduces nonlinear, multiscale dynamics that challenge traditional pharmacokinetic…

• Lipid nanoparticles (LNPs)
• mRNA therapeutics
• mathematical modeling
• multiscale modeling
• pharmacodynamics (pd)
• pharmacokinetics (pk)
• physiologically based pharmacokinetic (PBPK) modeling
• precision medicine
• quantitative systems pharmacology (QSP)
• translational modeling

108. 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

109. 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

110. Biomedical properties and emerging translational potential of bovine milk extracellular vesicles

Guan X, Hou X, Meng W, Guo Hd

Extracellular vesicles have garnered significant attention in the biomedical field due to their potential as therapeutic agents and diagnostic tools. Bovine milk extracellular vesicles (BMEVs), in particular, have emerged as a promising tool in biomedical research due to their unique biocompatibility, stability, and…

• Bovine milk extracellular vesicles
• CiteSpace
• Drug delivery
• Extracellular vesicle isolation
• Literature analysis
• Surface modification
• Targeted therapy

111. A Review of Osteosarcoma-Derived Exosomes in Tumor Progression, Chemoresistance, and Bone Regeneration, and Their Potential in Drug Delivery

Yan P, Yang Q, Jin A, Piao M

Exosomes, as critical mediators of intercellular communication, transport nucleic acids, proteins, and lipids that drive osteosarcoma (OS) progression and dynamically remodel the tumor microenvironment. Their distinctive biological properties-including low immunogenicity, high stability, and inherent targeting…

112. 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

113. 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

114. 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

115. 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…

116. 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

117. 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

118. 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

119. 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

120. 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…