Gene therapy: Comprehensive overview and therapeutic applications

N Sayed, P Allawadhi, A Khurana, V Singh, U Navik… - Life sciences, 2022 - Elsevier
Gene therapy is the product of man's quest to eliminate diseases. Gene therapy has three
facets namely, gene silencing using siRNA, shRNA and miRNA, gene replacement where …

The delivery of therapeutic oligonucleotides

RL Juliano - Nucleic acids research, 2016 - academic.oup.com
The oligonucleotide therapeutics field has seen remarkable progress over the last few years
with the approval of the first antisense drug and with promising developments in late stage …

Non-viral gene delivery systems for tissue repair and regeneration

P Wu, H Chen, R Jin, T Weng, JK Ho, C You… - Journal of translational …, 2018 - Springer
Critical tissue defects frequently result from trauma, burns, chronic wounds and/or surgery.
The ideal treatment for such tissue loss is autografting, but donor sites are often limited …

Modulating intracellular pathways to improve non-viral delivery of RNA therapeutics

T Van de Vyver, SC De Smedt, K Raemdonck - Advanced Drug Delivery …, 2022 - Elsevier
RNA therapeutics (eg siRNA, oligonucleotides, mRNA, etc.) show great potential for the
treatment of a myriad of diseases. However, to reach their site of action in the cytosol or …

A summary on non-viral systems for gene delivery based on natural and synthetic polymers

R Sarvari, M Nouri, S Agbolaghi… - … Journal of Polymeric …, 2022 - Taylor & Francis
Gene delivery systems necessitate the gene therapy, which is a unique approach for
applying the tunable genes to treat any diseases. To tackle down the disadvantages of viral …

Exosomes, extracellular vesicles and the eye

D Rudraprasad, A Rawat, J Joseph - Experimental Eye Research, 2022 - Elsevier
Exosomes are a subset of extracellular vesicles which accommodate a cargo of bioactive
biomolecules that generally includes proteins, nucleic acids, lipids, sugars, and related …

Effective silencing of ENaC by siRNA delivered with epithelial-targeted nanocomplexes in human cystic fibrosis cells and in mouse lung

AD Tagalakis, MM Munye, R Ivanova, H Chen… - Thorax, 2018 - thorax.bmj.com
Introduction Loss of the cystic fibrosis transmembrane conductance regulator in cystic
fibrosis (CF) leads to hyperabsorption of sodium and fluid from the airway due to …

Glycosylated triterpenoids as endosomal escape enhancers in targeted tumor therapies

H Fuchs, N Niesler, A Trautner, S Sama, G Jerz… - Biomedicines, 2017 - mdpi.com
Protein-based targeted toxins play an increasingly important role in targeted tumor
therapies. In spite of their high intrinsic toxicity, their efficacy in animal models is low. A major …

Delivery of ENaC siRNA to epithelial cells mediated by a targeted nanocomplex: a therapeutic strategy for cystic fibrosis

MDI Manunta, AD Tagalakis, M Attwood… - Scientific reports, 2017 - nature.com
The inhibition of ENaC may have therapeutic potential in CF airways by reducing sodium
hyperabsorption, restoring lung epithelial surface fluid levels, airway hydration and …

Food-derived peptides: Beneficial cns effects and cross-bbb transmission strategies

Z Li, Q Dang, P Wang, F Zhao, J Huang… - Journal of Agricultural …, 2023 - ACS Publications
Food-derived peptides, as dietary supplements, have significant effects on promoting brain
health and relieving central nervous system (CNS) diseases. However, the blood–brain …