Papers, explained in our own words

25. mRNA-LNPs encoding Ornithodoros moubata antigens induces humoral and cellular immune responses and tick resistance in rabbits

Cano-Argüelles Al, Vizcaíno-Marín R, González-Sánchez M, Oleaga A, Pérez-Sánchez R

Ornithodoros moubata and Ornithodoros erraticus are important vectors of African swine fever virus and relapsing fever Borrelia spp., highlighting the need for effective and sustainable control strategies. In this study, six previously identified O. moubata vaccine candidates, including salivary and/or midgut…

• Aquaporin
• Cross-protection
• Cytokine responses
• Ornithodoros moubata
• Subolesin
• mRNA-LNP vaccine
• vaccinomics

26. Gemcitabine Delivery and Nucleolin-Targeting Nanocarriers Based on N6L-Functionalized Polyelectrolyte Complexes and Layer-By-Layer Lipid Nanoparticles

Larue L, Brissault B, Chesneau C, Modjinou T, Leroy E, Hamadi S, Et Al.

Pancreatic ductal adenocarcinoma (PDAC) is highly resistant to chemotherapy due to dense stromal barriers. Although gemcitabine remains the standard treatment, its efficacy is limited. Combining it with the pseudopeptide N6L has shown synergistic effects, but administering them individually in biological systems…

• Polymer−gemcitabine conjugate
• gemcitabine
• layer-by-layer assembly
• nucleolin
• pancreatic ductal adenocarcinoma
• polyelectrolyte complexes

27. The blood-brain barrier and strategies for drug delivery to brain tumors

Nakhate V, Wen Py

Drug delivery across the blood-brain barrier (BBB) remains an important challenge in the treatment of brain tumors. This review summarizes the physiologic barriers to effective CNS drug exposure and evaluates strategies to improve delivery, with a focus on glioma. Recent advances span three complementary approaches…

• blood–brain barrier
• drug delivery
• focused ultrasound

28. An mRNA-encoded Bispecific T-Cell Engager Targeting Muscle-Specific Tyrosine Kinase for Myasthenia Gravis

Huang Y, Zhang J, Yu H, Zhang J, Zhao Y, Xu D, Et Al.

Muscle-specific tyrosine kinase antibody-positive myasthenia gravis (MuSK-MG) is a severe autoimmune disease with limited effective treatment options. We designed an mRNA-encoded bispecific T-cell engager (MuSK×CD3 TCE) comprising the human MuSK extracellular domain and an anti-CD3 single-chain variable fragment…

29. Extracellular vesicle-based drug delivery in pediatric immune thrombocytopenia

Di Domenico O, Marrapodi Mm, Di Feo G, Argenziano L, Rossi F, Di Paola A

Pediatric immune thrombocytopenia (ITP) is an acquired immune-mediated disorder triggered by autoantibody production directed against glycoproteins on the platelet surface, leading to platelet destruction in the spleen and impaired megakaryopoiesis in the bone marrow. The immune response is further compromised by a…

• cell-free therapy
• drug delivery
• extracellular vesicles
• immune thrombocytopenia
• immunomodulation
• mesenchymal stromal cells

30. Progress and applications of microneedle-based gene therapy

Shen Y, Ye X, Chen Q, Yu D, He L, Lei L, Et Al.

Gene therapy holds considerable promise for treating a wide range of intractable diseases through the modification of specific genes. However, gene therapeutics such as plasmid DNA, messenger RNA, and CRISPR-based systems are susceptible to degradation, face challenges in crossing cellular barriers, and may induce…

• Gene therapy
• Gene vectors
• Local delivery
• Microneedles
• Nucleic acid delivery

31. Comprehensive molecular characterization and biosafety assessment of TaDOF7.6 transgenic and gene-edited wheat

Gao S, Ullah U, Jin J, Shi Y, Zhang P, Zhang X, Et Al.

Wheat is one of the major staple food crops worldwide, and stable wheat yield is crucial for ensuring global food security. Transgenic and gene-editing technologies serve as important approaches for wheat genetic improvement, yet further application of related materials requires systematic evaluation of their…

• TaDOF7.6
• Environmental safety
• Gene-editing
• Genetic stability
• Molecular characteristics
• Transgene

32. Pomegranate Seed Oil Biologically Engineered Lactobacillus Extracellular Vesicles with Enhanced Yield and Brain Delivery via ApoE-Enriched Protein Corona

Duan D, Xu S, Wu K, Xu M, Zhou S, Yang X, Et Al.

The blood-brain barrier (BBB) is a major bottleneck for nanomedicine-based treatment of central nervous system (CNS) disorders. Lactobacillus -derived extracellular vesicles (LEV) are promising biocompatible nanocarriers, but their low production yield and limited BBB permeability hinder their development. We…

• Alzheimer’s disease
• Lactobacillus extracellular vesicles
• brain delivery
• membrane engineering
• pomegranate seed oil
• protein Corona

33. Advanced Nanocarriers for Delivering Traditional Chinese Medicine Monomers in Rheumatoid Arthritis: Prospects and Challenges

Yu H, He Z

Rheumatoid arthritis (RA) is an auto-immune disease that is accompanied by constant inflammation and degradation of joints, causing disabilities and poor quality of life. Even though the available therapies alleviate symptoms, long-term use is often linked with loss of effectiveness, systemic toxicity, and poor…

• bioavailability
• nanocarriers
• rheumatoid arthritis
• targeted drug delivery
• traditional chinese medicine

34. Are entirely virus-free CAR T cells as good as lentiviral transduced universal cells?

Gough O, Georgiadis C, Preece R, Kadirkamanathan R, Qasim W

Chimeric Antigen Receptor (CAR) T cells are commonly generated using gamma-retroviral or lentiviral vectors given their efficiency and stable integration, but vector supply constraints have created bottlenecks to wider access. Using a combination of lentiviral transduction and genome editing, 'universal' donor CAR-T…

35. Focused ultrasound-mediated AAV gene therapy: Unlocking a new space in the CNS delivery landscape

Todd N, Owusu-Yaw B, Hines E, Li Z, Mcdannold N, Maguire Ca, Et Al.

Adeno-associated viruses (AAVs) have emerged as the most clinically advanced platform for in vivo gene delivery, enabling durable therapeutic gene expression across a growing number of neurological disorders. However, successful delivery of AAVs to the brain requires navigating trade-offs among three interdependent…

36. Next-Generation Cancer Nanovaccines: Reprogramming the Tumor Microenvironment from Rational Design Toward Clinical Translation

Tabibzadeh S, Meng F, Asghariazar V, Safarzadeh E, Esmaeili A, Ten Hagen Tlm, Et Al.

Cancer vaccines have emerged as a promising immunotherapeutic strategy for inducing durable and tumour-specific immune responses; however, their clinical efficacy remains limited by poor antigen immunogenicity, inefficient delivery, tumor heterogeneity, and the highly immunosuppressive tumor microenvironment (TME).

• artificial intelligence
• cancer immunotherapy
• cancer nanovaccines
• precision oncology
• tumor microenvironment

37. Combining an autoactivated cGAS mutant with membrane-tethered IL-12 via mRNA lipid nanoparticles promotes systemic tumor control

Zhang A, Fang Y, Peng R, Yan M, Chen Z, Li T, Et Al.

Local tumor therapies rarely induce systemic immunity capable of controlling metastatic disease. Here, we engineer an mRNA lipid nanoparticle (LNP) platform to coordinate innate immune sensing and T cell activation in situ through co-delivery of an autoactivated cyclic GMP-AMP synthase mutant (cGAS R241E ) and…

38. CRISPR/Cas9-Mediated Targeting of the Integrin Beta-2 Gene as a Gene-Editing Strategy for Leukocyte Adhesion Deficiency Type I

Raji S, Soltaninejad H, Hosseinkhani S, Hamidieh Aa

Leukocyte adhesion deficiency (LAD) is a genetic immunodeficiency that severely impairs the leukocyte adhesion cascade and migration to the sites of infection and inflammation. As a result, the main consequence is recurrent and severe infections with bacteria and fungi, which can lead to death. The most common…

• CRISPR-Cas systems
• Cell-penetrating peptide
• Gene editing
• ITGB2 protein
• Leukocyte adhesion deficiency Type I

39. Hyaluronic acid-engineered NLCs as a macromolecular delivery platform for APOBEC3B siRNA and gemcitabine in triple-negative breast cancer: In vitro to in ovo assay

Quadir Ss, Tonge L, Ross K, Swadi R, Joshi G, Saleem I

Triple-negative breast cancer (TNBC) lacks targetable receptors and has poor clinical outcomes. This study combines hyaluronic acid (HA), a CD44-binding glycosaminoglycan, with APOBEC3B siRNA to engineer HA-functionalised nanostructured lipid carriers (HA-NLCs) that co-deliver gemcitabine hydrochloride (Gem HCl) and…

• APOBEC3B siRNA
• Breast cancer
• Chorioallantoic membrane (CAM) assay
• Gemcitabine hydrochloride
• Hyaluronic acid
• Nanostructured lipid carriers

40. Preclinical efficacy and safety of a novel lentiviral vector for ex vivo gene therapy of p47phox-deficient chronic granulomatous disease

Tsapogas P, Pastukhov O, Doerr V, Berger Ce, Chambovey M, Zala C, Et Al.

Chronic granulomatous disease is a primary immunodeficiency caused by mutations in the genes encoding subunits of the nicotinamide adenine dinucleotide phosphate oxidase complex, resulting in impaired antimicrobial activity of phagocytes. Patients suffer from recurrent infections and inflammation with…

41. Cell-penetrating peptide functionalized ZIF-90 platform for photodynamic-chemical combined therapy in colorectal cancer

Fu T, Ni B, Yin D, Zhu R, Liao S, Du Y, Et Al.

Colorectal cancer (CRC) is one of the major concerns of developed nations. The combination of different treatment modalities has been shown to be effective in improving tumor therapy efficiency and reducing side effects. However, the synergistic anti-tumor efficiency and highly responsive drug release still remain…

• ZIF-90
• cell-penetrating peptide
• chemotherapy
• colorectal cancer
• curcumin
• photodynamic therapy

42. Mosaic Hemagglutinin-Based Influenza B mRNA-LNP Vaccine Induces Strong Humoral Immune Response in Non-Human Primates

Vadovics M, Abbad A, Yellin T, Carreño Jm, Lam K, Muramatsu H, Et Al.

Developing broadly protective influenza vaccines remains a major challenge for global public health. Influenza B viruses cause about one-third of all annual influenza virus infections and current vaccines only show moderate effectiveness against matched virus strains, therefore, development of more effective and…

43. PINPOINT enables non-destructive nanoscopic quality control of individual lipid nanoparticles and their mRNA cargo

Banerjee P, Chatterjee T, Johnson-Buck A, Wrubel Ne, Ahnoud A, Walter Ng

Quality control of lipid nanoparticles (LNPs) is central to development of mRNA therapeutics, yet ensemble measurements obscure critical heterogeneity in particle morphology and mRNA cargo loading. Here we introduce PINPOINT (Profiling Individual Nanoparticles with Precise Observation of INternalized Therapeutics), a…

44. Microliter-scale in vitro aerosol-cell delivery of inhalable antisense oligonucleotide (ASO) induces "burst-like" pharmacokinetics

Kumar P, Chakraborty A, Richter E, Yang L, Schröppel A, Mergault C, Et Al.

Unlike traditional submerged cell cultures, nebulized drug delivery to air-liquid interface (ALI)-cultured lung cells provides a clinically relevant route for in vitro testing of inhalable drugs. Current nebulizer systems deliver non-physiologically large drug volumes. Here, we introduce the Cloud MAX technology…

• Cloud MAX technology
• Dosimetry
• Idiopathic pulmonary fibrosis (IPF)
• LIMCH1 gene silencing
• Precision aerosol-cell delivery
• Primary human lung fibroblasts

45. Hybrid Liposome-U87MG Membrane Nanovesicles Enhance Brain Delivery and Therapeutic Efficacy of Irinotecan in Orthotopic Glioma

Li S, Sun Y, Liu Y, An Q, Liu C, Cui H, Et Al.

The blood-brain barrier (BBB) and hydrolytic inactivation of the lactone form limit the efficacy of irinotecan (CPT-11) against glioma. To address these challenges, we developed a CPT-11-loaded hybrid liposome-U87MG cell membrane nanovesicle formulation (Hybrid/CPT-11), an underexplored biomimetic strategy for glioma…

• CPT-11
• biomimetic nanovesicles
• blood–brain barrier
• glioma
• hybrid nanovesicles
• irinotecan

46. Semi-randomized zwitterionic peptides: a breakthrough in drug delivery

Hernandez Pa, Moore Cj, Overby Ct, Benoit Dsw

Nanoparticle drug delivery systems (NP DDS) have transformed therapeutic delivery by improving drug stability, extending circulation time, and enabling targeted biodistribution. However, clinical translation remains limited by protein corona formation, immune recognition, and accelerated blood clearance following…

• immune recognition
• nanomedicine
• protein corona
• semi-randomized zwitterionic peptides
• zwitterionic peptides

47. In Vivo CRISPR Base Editing of ANGPTL3 for Durable Lipid Lowering

Mizoguchi T, Denizio Je, Chadwick Ac, Mazzola Am, Brown Mp, Mishra S, Et Al.

Circulating concentrations of low-density lipoprotein cholesterol (LDL-C) and triglyceride-rich lipoproteins are leading risk factors for atherosclerotic cardiovascular disease 1 . Despite several approved lipid-lowering medications, most patients fail to achieve target lipid levels under the chronic care treatment…

48. Disease-Specific Determinants of Cardiovascular Nanomedicine: Mechanisms, Limitations, and Translational Opportunities

Sivanathan B, Bobbala S, Venkatesh S

Cardiovascular disease (CVD) remains the leading cause of mortality worldwide, yet most therapies lack cardiovascular tissue- and cell-specificity, limiting efficacy and increasing off-target toxicity. Nanoparticle-based drug delivery systems offer opportunities to enhance myocardial targeting, improve…

• cardiomyocyte targeting
• drug delivery
• heart disease
• heart failure
• nanoparticle