Major degradable polycations as carriers for DNA and siRNA

Recent progress in gene therapy to deliver nucleic acids with multivalent cationic vectors

“…These were mainly directed to specific aspects of the compaction process, as the molecular characteristics of the hydrophobic regions and/or headgroups, and of the lipoplex structure [11,15,[20][21][22][23][24][25], the biochemical behavior [26][27][28][29][30], the structure-biological activity relationship [9,21,[31][32][33][34][35], or to several of them [7,8,10,13,36,37]. On the other hand, several reviews have been centered in polyplexes [14,[38][39][40][41] or in a wide variety of nanocarriers [16,17,[42][43][44]. The present review summarizes the biophysics and biochemistry studies carried on in the present decade with multivalent cationic gene vectors.…”

“…Its objective is to transfect nucleic acids (DNA or RNA) for curing human disease at a molecular level following one of these two possibilities: (i) repairing damaged cellular DNA by inserting and expressing appropriate DNA into the cells for performing the pre-designed activity [6][7][8][9][10][11][12][13][14][15]; or (ii) silencing the defective gene by inserting short interfering RNA (siRNA) to knockdown and stop the expression of the pathogenic protein [14,16,17]. The negative charge of plasmid DNA and RNA polyelectrolytes limits its transport through the cell membrane (also negatively charged) being necessary the use of gene vectors for compacting and transporting them into the cells.…”

Biodegradable and biocompatible cationic polymer delivering microRNA-221/222 promotes nerve regeneration after sciatic nerve crush

“…[17][18][19][20] Low molecular weight PEI (,20 kDa) has shown relatively low cytotoxicity but poor transfection efficiency. 21,22 Degradable linkers can conjugate with low molecular weight PEI to form cationic polymers with lower cytotoxicity and higher transfection efficiency. Lots of works have been done about cross-linked low molecular weight PEI in our previous research.…”

Insights into the therapeutic potential of hypoxia-inducible factor-1α small interfering RNA in malignant melanoma delivered via folate-decorated cationic liposomes

“…Although viral vectors have been proved efficacious, the safety concerns compromise their further application. In recent years, nonviral vectors have been extensively attempted to deliver siRNA, such as polyamidoamine, 11 poly(ethyleneimine), 12 and poly(amino acid). [13][14][15] Apart from poly(amino acid), the high toxicity of polymers limits their application in siRNA delivery.…”