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Papers, explained in our own words

Every entry summarises what the study set out to test, what it found and why it changes how we design delivery systems. Browse research and reviews or search the collection.

586 articles

Showing 1–6 of 6 articles · Keyword: gene therapy Clear keyword

Molecular pharmaceutics2026ResearchNon-viral Gene DeliveryDrug Delivery

1. 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…

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Seminars in hematology2026ResearchNon-viral Gene Delivery

2. Sickle cell disease-specific barriers to in vivo hematopoietic stem cell gene therapy

Choi Rh, Sheehan Va

Gene therapy for individuals living with sickle cell disease is a transformative therapy, ameliorating many of its severe symptoms. Nevertheless, ex vivo administration of gene therapy coupled with sickle cell disease specific obstacles, complicate and limit widespread adoption. In this review, we discuss current…

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Biotechnology journal2026ResearchNon-viral Gene Delivery

3. Comparison of Wildtype and Recombinant Adeno-Associated Virus (AAV) Production Reveals Distinct Productivity and Content Ratio Profiles

Alfieri S, Lee Kh

Recombinant adeno-associated virus is a leading viral vector for gene therapy. However, recombinant adeno-associated virus production systems have low titer and content ratio (ratio of genome-containing to total adeno-associated virus capsids), making it difficult to generate the large number of vector genomes…

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Molecular therapy. Nucleic acids2026ResearchNon-viral Gene DeliveryDrug Delivery

4. cGAS-mediated immunogenicity impairs the stability and safety of LNP-encapsulated DNA in vivo

Wang X, Liu T, Long B, Li M, Yao Y, Fu Y, Et Al.

DNA-based non-viral gene therapy offers a promising platform for titratable gain-of-function treatments, enabling multi-dosing and sustained efficacy. However, its clinical translation has been constrained by unintended immune activation. We systematically investigated the pathogenesis of DNA drug-induced toxicity in…

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ACS nanoscience Au2026ResearchNon-viral Gene DeliveryDrug Delivery

5. Lipid Nanoparticles at the Crossroads of Chemistry and Medicine: A Tutorial

Rogado Mt, Wang A, Zeng S, Wang Z, Burda C

Lipid nanoparticles (LNPs) have risen to the forefront of modern therapeutics by enabling the clinical success of COVID-19 mRNA vaccines. In order for them to function as drug delivery vesicles, LNPs must have scalable, tunable, and targeted nanoscale platforms whose strengths in versatility, performance, and…

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Journal for immunotherapy of cancer2026ResearchNon-viral Gene DeliveryDrug Delivery

6. IL7R mRNA therapy enhances antitumor T cell immunity and suppresses tumor growth in preclinical tumor models

Rehman Au, Khan Mn, Akhtar A, Qin Q, Zhang X, Asghar Ma, Et Al.

Interleukin-7 receptor (IL7R) signaling is critical for T-cell survival, homeostasis, and immune function, but the therapeutic potential of increasing IL7R expression through messenger RNA (mRNA) delivery remains poorly defined. We investigated whether IL7R mRNA therapy could enhance antitumor immunity and improve…

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