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.
706 articles
Showing 25–48 of 86 articles · Keyword: siRNA Clear keyword
Karen Bulaklak And Charles A. Gersbach
Gene therapy is at an inflection point: recent approvals and clinical successes have validated the field, but broader application is still limited by immune responses to vectors, inefficient delivery outside the retina and liver, and regulatory frameworks that are poorly suited to personalised, potentially curative…
Read the articleFrancia V, Schiffelers Rm, Cullis Pr, Witzigmann D
LNPs are clinically advanced vectors for siRNA, mRNA, and DNA delivery, but their in vivo behaviour is strongly influenced by the biological environment. Upon administration, biomolecules adsorb to the LNP surface and form a “biomolecular corona,” which defines the particle’s biological identity, affects stability…
Read the articleLiu C, Zhang L, Dong N, Zhang W, Chen X, Gao R, Sun H.
Cationic liposomes (CLs) are promising non-viral gene-delivery vectors, but their clinical use in cancer gene therapy is limited by extracellular barriers (opsonisation, RES clearance, poor tumour penetration) and intracellular barriers (endosomal/lysosomal entrapment, restricted cytoplasmic/nuclear transport). The…
Read the articleNina Filipczak, Jiayi Pan, Satya Siva Kishan Yalamarty, Vladimir P. Torchilin
Liposomes remain an important nano-sized drug delivery system because of their biomimetic bilayer, ease of preparation, and biocompatibility, but clinical translation is still limited by reproducibility, scale-up, stability, and payload-specific barriers. The review updates recent advances in liposome preparation…
Read the articleChen, C.-K.; Huang, P.-K.; Law, W.-C.; Chu, C.-H.; Chen, N.-T.; Lo, L.-W
Gene therapy is limited by the fragile nature of therapeutic DNA/RNA and by safety concerns with viral vectors. Non-viral polymeric vectors are attractive, but conventional cationic polymers such as PEI, PDMAEMA, and PLL are often non-degradable, causing accumulation, cytotoxicity, and poor gene unpacking. This…
Read the articleWahane A, Waghmode A, Kapphahn A, Dhur K, Gupta A, Bahal R
Nucleic acid analogs and genome-editing proteins have broad therapeutic potential, but clinical translation is limited by poor cell- and organ-specific delivery, endosomal entrapment, enzymatic degradation, and short bioavailability. The review examines lipid- and polymer-based non-viral vectors as strategies to…
Read the articlePiotr S. Kowalski, Arnab Rudra, Lei Miao, Daniel G. Anderson
mRNA has broad therapeutic potential for vaccination, protein replacement, and gene editing, but its clinical translation is limited by the need for improved intracellular delivery systems. mRNA is much larger than siRNA or ASOs (~300–5,000 kDa, ~1–15 kb vs ~14 kDa and 4–10 kDa, respectively), making delivery more…
Read the articleMajumder J, Et Al.
Conventional systemic drug administration suffers from low site-specific bioavailability, unfavourable biodistribution, poor target accumulation, and adverse side effects. There is a need for carrier-based systems that enable localised and targeted delivery, improve therapeutic efficacy, and reduce toxicity…
Read the articleZhao W, Hou X, Vick Og, Dong Y.
Genetic and rare diseases (GARDs) affect more than 350 million patients worldwide, and many lack effective treatments. RNA-based therapeutics—siRNA, mRNA, miRNA, lncRNA, and RNA-containing genome editing systems—are promising, but naked RNA has poor cellular penetration, is degraded in biological fluids, and does not…
Read the articleRun Ni, Ruoyu Fang, And Ying Chau
While many reviews have systematically covered different types of formulation materials for gene delivery, a parallel comparison of different formulation materials together with an analysis of the correlation between their material properties and nucleic acid delivery functions has not been conducted. Understanding…
Read the articleSamuel A. Smith, Laura I. Selby, Angus P. R. Johnston, And Georgina K. Such
Many emerging therapies require delivery of biological cargo into the cytosol, but nanoparticle delivery systems are hindered by entrapment and degradation in the endo/lysosomal pathway. Endosomal escape remains a rate-determining step for non-viral delivery. The review highlights the need for greater understanding…
Read the articleScheideler M, Vidakovic I, Prassl R
miRNAs can regulate about 60% of protein-coding genes and enable targeting of previously “undruggable” proteins and diseases, but their clinical use is limited by nuclease degradation, poor tissue targeting, inefficient cellular uptake, and inadequate cytosolic release. Lipid nanocarriers are a versatile…
Read the articleSima Rezvantalab And Mostafa Keshavarz Moraveji
PLGA is a biocompatible, biodegradable copolymer widely used in drug delivery systems (DDS), but conventional bulk synthesis methods suffer from poor control over particle size, broad size distribution, low drug loading/encapsulation efficiency, and burst release. Microfluidic systems offer a tightly controlled…
Read the articleNot Fully Listed In The Supplied File; Corresponding Authors Indicated By Emails: [email protected] And [email protected] Doi/link: Not Included In The Supplied File
Endosomal escape remains a major roadblock for delivering biological therapeutics such as DNA, siRNA, and proteins. Polymeric nanoparticles can protect cargo and target cells, but they often remain trapped in endosomal/lysosomal compartments, where cargo is degraded. A better understanding of how nanoparticle…
Read the articleYao Xiao, Kun Shi, Ying Qu, Bingyang Chu, Zhiyong Qian
In the past 10 years, many clinical nano-gene therapy trials have been discontinued due to poor efficacy and serious side effects. There is a critical need to design suitable gene delivery systems that can protect nucleic acids, enhance transfection efficiency, penetrate deep into tumours, and remain stable and safe…
Read the articleIrene Lostalé-Seijo And Javier Montenegro
Gene therapy has enormous therapeutic potential, but clinical translation is limited by the delivery problem: nucleic acids are negatively charged, unstable, and must overcome multiple biological barriers to reach their intracellular site of action. Viral vectors are efficient but suffer from immunogenicity, limited…
Read the articleEvers Mjw, Kulkarni Ja, Van Der Meel R, Cullis Pr, Vader P, Schiffelers Rm
Free siRNA, mRNA, pDNA, and CRISPR/Cas9 components are rapidly degraded and cleared, so delivery systems are required. LNPs are the most clinically advanced non-viral siRNA carriers; Patisiran reached Phase III and market application in late 2017. The field needed a clearer understanding of LNP design parameters and…
Read the articleHassan Dianat‐moghadam, Maryam Heidarifard, Rana Jahanban‐esfahlan, Yunes Panahi, Hamed Hamishehkar, Farhad Pouremamali, Reza Rahbarghazi, Mohammad Nouri
Cancer stem cells (CSCs) drive tumour heterogeneity, drug resistance, recurrence, and metastasis, limiting conventional chemo/radiotherapy. Liposomal drug delivery systems (LDDSs) are biocompatible, versatile nanocarriers that may overcome CSC-mediated resistance, but their clinical translation remains limited by…
Read the articleKarlsson, J.; Vaughan, H. J.; Green, J. J
Biodegradable polymeric nanoparticles have great potential to improve cancer therapy by enabling targeted delivery, triggered release, and reduced off-target toxicity. This review addresses how such nanocarriers can be engineered to overcome systemic, organ-level, and cellular barriers and to target cancer cells…
Read the articleTzeng Sy, Green Jj
Nucleic acids can be used to engineer the immune system, but their clinical use requires better specificity and intracellular delivery efficiency. Viral vectors have manufacturing, cargo-capacity, safety, and immunological drawbacks; therefore, polymers are being developed as non-viral delivery vehicles whose…
Read the articleSima Rezvantalab, Natascha Ingrid Drude, Mostafa Keshavarz Moraveji, Nihan Güvener, Emily Kate Koons, Yang Shi, Twan Lammers, Fabian Kiessling
PLGA-based nanoparticles are widely investigated for cancer therapy, but their clinical translation remains limited. A consolidated overview was needed of how PLGA NP physicochemical properties, biological behaviour, targeting strategies, and combination therapies interact—and why promising preclinical results have…
Read the articleHaiqing Bai, Gillian M. Schiralli Lester, Laura C. Petishnok, David A. Dean
Productive gene transfer requires plasmid DNA to not only enter cells but also traffic through the cytoplasm and cross the nuclear envelope. Most transfection strategies focus on membrane entry and promoter choice, but cytoplasmic trafficking and nuclear import are critical, rate-limiting barriers—especially because…
Read the articleJiménez Blanco Jl, Benito Jm, Ortiz Mellet C, García Fernández Jm.
Nonviral gene delivery lacks molecularly well-defined carriers suitable for rigorous structure–activity relationship (SAR) studies. Viral vectors raise immunogenicity, cost, and cargo-size concerns, while polymers, dendrimers, and cationic lipids are often polydisperse or supramolecularly ill-defined. The review…
Read the articleMizrahy S, Hazan-Halevy I, Landesman-Milo D, Ng Bd, Peer D
Current cancer immunotherapy shows promise but fails across many tumour types, likely because single-pathway treatments cannot overcome the complex immunosuppressive tumour microenvironment (TME). Lipid-based nanoparticles (LNPs) may allow simultaneous, temporally coordinated delivery of multiple immune modulators to…
Read the articleWe co-develop nanocarrier and biosensing programmes with pharma, biotech and academic groups — from target selection through scale-up.
