Human gene therapy2026ResearchNon-viral Gene Delivery
Bacha T, Ren Y, Fan J, Mcmahon Mm, Panigrahi A, Warner D, Et Al.
Adeno-associated virus (AAV) vectors for gene delivery have demonstrated efficacy in treating genetic and acquired human diseases. For the treatment of neurological disorders, AAV delivery via the cerebrospinal fluid (CSF) enables transduction of therapeutic genes to cellular targets in the nervous system, bypassing…
Read the article →Cancer drug resistance (Alhambra, Calif.)2026ResearchNon-viral Gene DeliveryDrug Delivery
Buscail L, Lebrin M, Hanoun N, Gourgou S, Dorat N, Vernejoul F, Et Al.
Aim: Pancreatic ductal adenocarcinoma (PDAC) has a dismal prognosis, and innovative therapies are needed. We developed a phase I pilot, non-viral gene therapy (CYL-02) encoding SST2 and DCK::UMK genes complexed with a polyethyleneimine non-viral vector to sensitize tumours to gemcitabine chemotherapy, which showed…
Read the article →Molecular pharmaceutics2026ResearchNon-viral Gene DeliveryDrug Delivery
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…
Read the article →Seminars in hematology2026ResearchNon-viral Gene Delivery
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…
Read the article →Biotechnology journal2026ResearchNon-viral Gene Delivery
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…
Read the article →Molecular therapy. Nucleic acids2026ResearchNon-viral Gene DeliveryDrug Delivery
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…
Read the article →ACS nanoscience Au2026ResearchNon-viral Gene DeliveryDrug Delivery
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…
Read the article →Journal for immunotherapy of cancer2026ResearchNon-viral Gene DeliveryDrug Delivery
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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