Current cardiology reports2026ReviewNon-viral Gene Delivery
Gunes Ea, Patel U, Iyer S, Taub A, Lee-Guest E, Cline S, Et Al.
Danon disease is a rare, highly penetrant and morbid X-linked cardioskeletal myopathy caused by loss-of-function variants in the lysosome-associated membrane protein 2 (LAMP2) gene. We review the rationale for LAMP2B gene replacement, the basis for adeno-associated virus serotype 9 (AAV9) vector selection, the…
Read the article →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 →Annals of allergy, asthma & immunology : official publication of the American College of Allergy, Asthma, & Immunology2026ReviewNon-viral Gene Delivery
Schauberger Em, Phillips Ja
The management of allergic and immunologic diseases has long centered on symptom management rather than disease modification. Conventional pharmacotherapy controls manifestations of the immune dysregulation but does little to treat the underlying cause. Newer monoclonal and small molecule therapies target downstream…
Read the article →MedComm2026ReviewNon-viral Gene DeliveryDrug Delivery
Ma X, Li L, Sun Y
CRISPR-Cas genome editing represents one of the most transformative advances in modern molecular biology, enabling programmable site-specific genetic modification that has revolutionized functional genomics and precision therapeutic development. Beyond canonical Cas9 nucleases, the CRISPR toolkit has diversified into…
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 →Materials today. Bio2026ReviewNon-viral Gene Delivery
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…
Read the article →Molecular medicine reports2026ReviewNon-viral Gene Delivery
Qin R, Hu X, Jiang W, Wang J, Zheng M, Zhang Z, Et Al.
Genetic epilepsy, for which the molecular mechanisms are relatively well defined in numerous monogenic forms, represents an attractive model for the transition from symptomatic seizure control to mechanism‑driven correction. However, gene therapy strategies differ in their mechanisms of action, adaptability to…
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 →Nanomedicine (London, England)2026ReviewNon-viral Gene Delivery
Thakur Sk, Malviya R, Chandra P, Sridhar Sb, Shareef J, Wadhwa T
Choroidal melanoma is the most common primary intraocular malignancy, and effective gene therapy remains limited by ocular barriers, tumor heterogeneity, and inefficient nucleic acid delivery. This review critically analyses the molecular rationale, engineering principles, emerging nanoarchitectures, biological…
Read the article →Journal of orthopaedic translation2026ReviewNon-viral Gene DeliveryDrug Delivery
Chen X, Sun J, Xie W, Arthur Vithran Dt, Zhang Q, Rosenholm Jm, Et Al.
Degenerative musculoskeletal diseases (DMDs) are a group of chronic conditions characterized by the progressive deterioration of tissue structure and function, primarily affecting the articular cartilage, bones, tendons, and ligaments. With global aging populations, the rising prevalence of these disorders presents a…
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 →Molecular therapy. Advances2026ReviewNon-viral Gene Delivery
Keating Al, Carlisle Rc, Mitrophanous Ka, Seymour Lw, Fisher Kd
Lentiviral vector (LVV)-mediated chimeric antigen receptor (CAR)-T cell therapy has transformed the landscape for blood cancer treatment through genetic modification of patients' T cells. Yet, the complexity of the manufacturing process makes this therapy inaccessible for many patients. Systemic intravenous delivery…
Read the article →Molecular therapy. Oncology2026ReviewNon-viral Gene DeliveryDrug Delivery
Bedada At, Genet S
Cancer remains a formidable global health challenge, representing the second leading cause of mortality worldwide. In 2023, there were approximately 18.5 million new cancer cases and over 10 million deaths globally, with projections estimating 30.5 million new cases and 18.6 million deaths annually by 2050. In the…
Read the article →Viruses2026ReviewNon-viral Gene Delivery
Zhang M, Mohess J, Cheung J, Du Q, Qiu J, Wang J
Osteoarthritis (OA) is the most prevalent joint disease, which is pathologically characterized by articular cartilage degradation, chronic synovial inflammation, subchondral bone remodeling, and osteophyte formation. Patients with OA suffer from joint pain, deformity, and functional impairment. Current therapies…
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 →Discover genetics and evolution2026ReviewNon-viral Gene Delivery
Bhardwaj A
Off-target activity remains a significant challenge in the clinical development of CRISPR-based therapies. As programmable nucleases advance from experimental tools toward approved medicines, the ability to predict, detect, and mitigate unintended genomic editing events has acquired direct translational importance.
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…
Read the article →Advances in Colloid and Interface Science2026ReviewNon-viral Gene DeliveryDrug Delivery
D Zhi, A Halemaimaiti, B Dai, H Li, H Wang, Z Zhang, J Yu, S Zhang
This review examines how lipid structure and formulation affect the delivery of DNA, mRNA and siRNA by lipid nanoparticles, while assessing tissue targeting, safety and clinical translation challenges.
Read the article →BioDrugs2026ReviewNon-viral Gene DeliveryDrug Delivery
U Iqbal, Rw Hwang, Wj Costain
A review of blood–brain barrier transport mechanisms and emerging lipid nanoparticle strategies for carrying nucleic acid and other complex therapies into the central nervous system.
Read the article →Molecular Therapy Methods & Clinical Development2025ReviewNon-viral Gene Delivery
Ca Brimacombe, Ja Kulkarni, Mhy Cheng, K An, D Witzigmann, Pr Cullis
A review of the physical principles and development history behind siRNA lipid nanoparticles for liver gene silencing and mRNA lipid nanoparticles for protein expression.
Read the article →Nature Reviews Drug Discovery2024ReviewNon-viral Gene DeliveryDrug Delivery
Pr Cullis, Pl Felgner
This historical review traces lipid-based gene delivery from cationic lipoplexes to ionisable lipid nanoparticles, connecting foundational research with approved mRNA vaccines and siRNA therapy.
Read the article →