| Title |
Cationic polyelectrolyte-mediated delivery of antisense morpholino oligonucleotides for exon-skipping in vitro and in mdx mice
|
|---|---|
| Published in |
International Journal of Nanomedicine, September 2015
|
| DOI | 10.2147/ijn.s89910 |
| Pubmed ID | |
| Authors |
Mingxing Wang, Bo Wu, Jason D Tucker, Peijuan Lu, Qilong Lu |
| Abstract |
In this study, we investigated a series of cationic polyelectrolytes (PEs) with different size and composition for their potential to improve delivery of an antisense phosphorodiamidate morpholino oligomer (PMO) both in vitro and in vivo. The results showed that the poly(diallyldimethylammonium chloride) (PDDAC) polymer series, especially PE-3 and PE-4, improves the delivery efficiency of PMO, comparable with Endoporter-mediated PMO delivery in vitro. The enhanced PMO delivery and targeting to dystrophin exon 23 was further observed in mdx mice, up to fourfold with the PE-4, compared with PMO alone. The cytotoxicity of the PEs was lower than that of Endoporter and polyethylenimine 25,000 Da in vitro, and was not clearly detected in muscle in vivo under the tested concentrations. Together, these results demonstrate that optimization of PE molecular size, composition, and distribution of cationic charge are key factors to achieve enhanced PMO exon-skipping efficiency. The increased efficiency and lower toxicity show this PDDAC series to be capable gene/antisense oligonucleotide delivery-enhancing agents for treating muscular dystrophy and other diseases. |
X Demographics
The data shown below were collected from the profiles of 6 X users who shared this research output. Click here to find out more about how the information was compiled.
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| United States | 2 | 33% |
| Unknown | 4 | 67% |
Demographic breakdown
| Type | Count | As % |
|---|---|---|
| Members of the public | 3 | 50% |
| Practitioners (doctors, other healthcare professionals) | 2 | 33% |
| Scientists | 1 | 17% |
Mendeley readers
The data shown below were compiled from readership statistics for 17 Mendeley readers of this research output. Click here to see the associated Mendeley record.
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| Sweden | 1 | 6% |
| Unknown | 16 | 94% |
Demographic breakdown
| Readers by professional status | Count | As % |
|---|---|---|
| Student > Ph. D. Student | 3 | 18% |
| Student > Doctoral Student | 2 | 12% |
| Researcher | 2 | 12% |
| Student > Bachelor | 1 | 6% |
| Professor | 1 | 6% |
| Other | 3 | 18% |
| Unknown | 5 | 29% |
| Readers by discipline | Count | As % |
|---|---|---|
| Agricultural and Biological Sciences | 3 | 18% |
| Biochemistry, Genetics and Molecular Biology | 2 | 12% |
| Neuroscience | 2 | 12% |
| Medicine and Dentistry | 1 | 6% |
| Chemistry | 1 | 6% |
| Other | 1 | 6% |
| Unknown | 7 | 41% |
Attention Score in Context
This research output has an Altmetric Attention Score of 4. This is our high-level measure of the quality and quantity of online attention that it has received. This Attention Score, as well as the ranking and number of research outputs shown below, was calculated when the research output was last mentioned on 11 September 2016.
All research outputs
#7,959,659
of 25,371,288 outputs
Outputs from International Journal of Nanomedicine
#940
of 4,121 outputs
Outputs of similar age
#86,542
of 276,785 outputs
Outputs of similar age from International Journal of Nanomedicine
#31
of 154 outputs
Altmetric has tracked 25,371,288 research outputs across all sources so far. This one has received more attention than most of these and is in the 67th percentile.
So far Altmetric has tracked 4,121 research outputs from this source. They receive a mean Attention Score of 4.7. This one has done well, scoring higher than 75% of its peers.
Older research outputs will score higher simply because they've had more time to accumulate mentions. To account for age we can compare this Altmetric Attention Score to the 276,785 tracked outputs that were published within six weeks on either side of this one in any source. This one has gotten more attention than average, scoring higher than 67% of its contemporaries.
We're also able to compare this research output to 154 others from the same source and published within six weeks on either side of this one. This one has done well, scoring higher than 79% of its contemporaries.