Nature Reviews Drug Discovery2021ReviewNon-viral Gene Delivery
Chaudhary N, Weissman D, Whitehead Ka.
mRNA vaccines have progressed from a scepticism-inducing idea to clinical reality, with COVID-19 catalysing the fastest vaccine development in history. Remaining needs include optimising mRNA design, intracellular delivery, and applications beyond SARS-CoV-2 prophylaxis. This review describes mRNA vaccine…
Read the article →Nature Nanotechnology2021ReviewNon-viral Gene Delivery
Gong N, Sheppard Nc, Billingsley Mm, June Ch, Mitchell Mj.
T-cell-based immunotherapies have shown clinical success in B-cell malignancies, but broad implementation is limited by insufficient T-cell expansion, poor trafficking into solid tumours, T-cell exhaustion in hostile tumour microenvironments, and loss of target antigen expression. Nanomaterials may uniquely overcome…
Read the article →2021ReviewDrug Delivery
Venkatas J, Singh M.
Cervical cancer is immunologically relevant because retained high-risk HPV antigens can be recognised as foreign, making immunotherapy attractive. However, conventional immunotherapy is limited by variable efficacy, immune evasion, cytokine toxicity, and poor targeting. Nanomedicine may improve delivery, targeting…
Read the article →Seminars in Immunology2021ReviewNon-viral Gene Delivery
Est-Witte Se, Livingston Nk, Omotoso Mo, Green Jj, Schneck Jp.
T cell therapies (TIL, TCR, CAR T) show clinical efficacy but are limited by high cost, lengthy ex vivo manufacturing, patient variability, and poor persistence. Nanoparticles offer a modular, universal platform to improve T cell therapy at every stage—from antigen presentation and T cell activation to T cell…
Read the article →Advanced Healthcare Materials2021ReviewNon-viral Gene Delivery
Ho W, Gao M, Li F, Li Z, Zhang X-Q, Xu X.
Nucleic acid vaccines (DNA and mRNA) offer rapid design, adaptability to changing pathogen strains, and stimulation of both humoral and cellular immunity, but their clinical translation is limited by inefficient delivery—nucleic acids are rapidly degraded by endogenous nucleases, have poor cellular uptake due to…
Read the article →Advanced Drug Delivery Reviews2021ReviewNon-viral Gene Delivery
Kim J, Eygeris Y, Gupta M, Sahay G.
mRNA vaccines are promising but naked mRNA is fragile, susceptible to enzymatic degradation, poorly taken up by cells due to electrostatic repulsion, and can trigger innate immune responses. Self-assembly offers a versatile approach to prepare delivery vehicles with customisable properties. The review discusses…
Read the article →Nature Communications2021ReviewNon-viral Gene Delivery
Han X, Zhang H, Butowska K, Swingle Kl, Alameh M-G, Weissman D, Mitchell Mj
RNA therapeutics—ASOs, siRNAs, miRNAs, mRNAs, and CRISPR-Cas9 sgRNAs—are limited by nuclease degradation, large size, and negative charge. Ionisable lipids are the key LNP component that condenses RNA, enables endosomal escape, and reduces toxicity. Since 2008, many ionisable lipids have been created, but the field…
Read the article →Bioengineering & Translational Medicine2021ReviewNon-viral Gene Delivery
Schlich M, Palomba R, Costabile G, Mizrahy S, Pannuzzo M, Peer D, Decuzzi P
Endosomal escape remains the major intracellular barrier for RNA therapeutics. Only a small fraction of RNA delivered by ionisable lipid nanoparticles (LNPs) reaches the cytosol, where siRNA, miRNA, mRNA, and CRISPR components must act. A deeper mechanistic understanding is needed to design next-generation LNPs with…
Read the article →Trends in Molecular Medicine2021ReviewNon-viral Gene Delivery
Likely Citation: Swingle Kl, Hamilton Ag, Mitchell Mj
mRNA is degraded by nucleases and cannot easily cross cell membranes because of its large size and negative charge. Delivery therefore requires encapsulation in vehicles such as lipid nanoparticles (LNPs) to enable protein replacement, vaccines, and gene-editing applications. Onpattro was the first FDA-approved…
Read the article →Nature Reviews Materials2021ReviewNon-viral Gene Delivery
Hou X, Zaks T, Langer R, Dong Y
mRNA has therapeutic potential for vaccines, protein replacement, cancer immunotherapy, cellular reprogramming, and genome editing, but it requires safe, effective, and stable delivery systems to protect it from degradation and enable cellular uptake and mRNA release. Lipid nanoparticles have entered the clinic for…
Read the article →Pharmaceutics2021ReviewNon-viral Gene Delivery
Zak Mm, Zangi L
mRNA therapeutics are limited by innate immune activation, rapid RNase degradation, and inefficient delivery to target organs. Although LNPs are the only clinically approved RNA therapeutic carriers, most systemically delivered LNPs accumulate in the liver, so organ-specific delivery remains a major barrier for…
Read the article →ACS Nano.2021ReviewNon-viral Gene Delivery
Rumiana Tenchov, Robert E. Bird, Allison E. Curtze, Qiongqiong Angela Zhou
Lipid nanoparticles (LNPs) have become key delivery vehicles across pharmaceuticals, with renewed prominence as the delivery platform for COVID-19 mRNA vaccines. The field lacks a broad, quantitative landscape of LNP research across liposomes, solid lipid nanoparticles, nanostructured lipid carriers, cationic…
Read the article →Journal of Drug Delivery Science and Technology2021ReviewNon-viral Gene Delivery
Ashok B, Peppas Na, Wechsler Me
CRISPR/Cas9 genome editing holds major therapeutic promise, but in vivo delivery is arguably the most difficult barrier to clinical translation. Efficient, selective transport of editing components to target cells/tissues while minimising off-target effects remains unresolved. This review critiques lipid- and…
Read the article →2020ReviewNon-viral Gene Delivery
Zeb A, Rana I, Choi H-I, Et Al.
Biopharmaceuticals have become clinically and commercially important due to their specificity, potency, targeting ability, and reduced side effects, but their macromolecular structure and intrinsic instability make formulation and administration challenging; parenteral delivery remains the only viable option in most…
Read the article →Frontiers in Chemistry2020ReviewNon-viral Gene Delivery
Guevara Ml, Persano F, Persano S
mRNA is a flexible and potentially safer cancer immunotherapy platform, but its clinical use has been limited by extracellular instability, poor cellular uptake, and inefficient endosomal escape. Lipid nanoparticles have become the most advanced non-viral delivery system for mRNA, enabling therapeutic vaccines…
Read the article →Bioconjugate Chemistry2020ReviewNon-viral Gene Delivery
Francia 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 article →Advanced Drug Delivery Reviews2020ReviewDrug Delivery
Nina 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 article →Expert Opinion on Drug Delivery2020ReviewNon-viral Gene Delivery
Karlsson, J.; Rhodes, K. R.; Green, J. J.; Tzeng, S. Y
Non-viral gene delivery needs safe and efficient carriers. PBAEs are biodegradable cationic polymers with promising transfection activity, but they face challenges such as instability in physiological fluids, positive-charge toxicity, and limited systemic delivery. This review summarises PBAE properties, advantages…
Read the article →Pharmaceutics2020ReviewNon-viral Gene Delivery
Wahane 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 article →Molecular Therapy2019ReviewNon-viral Gene Delivery
Piotr 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 article →Cancer Letters2019ReviewNon-viral Gene Delivery
Mukalel Aj, Riley Rs, Zhang R, Mitchell Mj.
Nucleic acid therapeutics—DNA, mRNA, and CRISPR/Cas9 genome editing systems—hold significant potential for cancer immunotherapy but face major delivery barriers, including rapid in vivo degradation, poor cellular uptake, required nuclear entry, and toxicity in healthy tissues. Nanoparticle delivery systems are being…
Read the article →Biomaterials2019ReviewNon-viral Gene Delivery
Zhao 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 article →Journal of Pharmaceutical Investigation2019ReviewDrug Delivery
PLA and PLGA are FDA-approved, biodegradable polymers widely used for micro- and nanoparticle drug delivery, but their clinical performance depends on biocompatibility, degradation behaviour, and drug-release control. This review addresses the need to understand PLA/PLGA tissue responses, degradation pathways…
Read the article →Therapeutic Delivery* (Future Science Group) Year/Publication: Published online 4 December 20182018ReviewNon-viral Gene Delivery
Not 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…
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