Aim To avoid the limitation of the use of cationic polyethlenimine (PEI)-complexed plasmid DNA use for in vitro or in vivo gene delivery due to its cytotoxicity and lower efficiency in the presence of serum. Methods A polyplex with decreased positive charge on the complex surface was designed. The PEI/DNA (PD) complexes coated with an anionic biodegradable polymer, alginate were prepared and their gene delivery behavior with PD was compared. Results The alginate-coated PD polyplex, where alginate: PEI: DNA [alginate: DNA, 0. 15 (w/w); PEI: DNA, N: P = 10] showed about 10 -30 fold-increased transfection efficiency compared to corresponding non-coated complexes to C3 cells in the presence of 50% serum. The surface charge of the alginate-coated complex was approximately half of that of the alginate-lacking complex. The size of alginate-coated complex was slightly smaller than that of the corresponding complex without alginate. The former complex also showed a reduced erythrocyte aggregation activity and decreased cytotoxicities to C3 cells in comparison with PD complex. Conclusion The alginate-coated PD polyplexes as a new gene delivery system can improve transfection efficiency in high serum concentration with low cytotoxicity to C3 cells.

PURPOSE: To evaluate the association of apolipoprotein E (APOE) polymorphism and cataracts in the Korean population. METHODS: The present research included participants from a population-based study in Incheon, Korea. A sample of 126 adults genotyped for polymorphisms of APOE underwent a medical interview, an eye examination which included visual acuity testing, slitlamp cataract evaluation and fundus examination. The APOE polymorphism was determined using a polymerase chain reaction method. RESULTS: Eighty-eight participants (69.8%) were diagnosed with cataracts or had undergone cataract surgery in 1 or both eyes, and 38 participants (30.2%) demonstrated no signs of cataract. The frequencies of the APOE genotypes and alleles were not significantly different from the cataract and the control group. APOE epsilon2 carriers were less likely to have cataracts than non-epsilon2 carriers with an odds ratio of 0.367 which was almost statistically significant with the multiple logistic regression analysis (p = 0.052). CONCLUSIONS: There was no significant correlation of APOE genotype and cataracts. However, a slight negative association of APOE epsilon2 and cataracts were found in the Korean population.
Adult ; Alleles ; Apolipoprotein E2 ; Apolipoproteins ; Apolipoproteins E ; Cataract ; Eye ; Genotype ; Humans ; Korea ; Logistic Models ; Odds Ratio ; Polymerase Chain Reaction ; Visual Acuity

OBJECTIVE: Huntington's disease (HD) is a genetic neurodegenerative disease that is caused by abnormal CAG expansion. Altered microRNA (miRNA) expression also causes abnormal gene regulation in this neurodegenerative disease. The delivery of abnormally downregulated miRNAs might restore normal gene regulation and have a therapeutic effect. METHODS: We developed an exosome-based delivery method to treat this neurodegenerative disease. miR-124, one of the key miRNAs that is repressed in HD, was stably overexpressed in a stable cell line. Exosomes were then harvested from these cells using an optimized protocol. The exosomes (Exo-124) exhibited a high level of miR-124 expression and were taken up by recipient cells. RESULTS: When Exo-124 was injected into the striatum of R6/2 transgenic HD mice, expression of the target gene, RE1-Silencing Transcription Factor, was reduced. However, Exo-124 treatment did not produce significant behavioral improvement. CONCLUSION: This study serves as a proof of concept for exosome-based delivery of miRNA in neurodegenerative diseases.
Animals ; Cell Line ; Exosomes ; Huntington Disease* ; Methods ; Mice ; MicroRNAs ; Neurodegenerative Diseases ; Transcription Factors