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Attention Score in Context
| Title |
Optimization of trans-Splicing for Huntington's Disease RNA Therapy
|
|---|---|
| Published in |
Frontiers in Neuroscience, October 2017
|
| DOI | 10.3389/fnins.2017.00544 |
| Pubmed ID | |
| Authors |
Hansjörg Rindt, Colton M. Tom, Christian L. Lorson, Virginia B. Mattis |
| Abstract |
Huntington's disease (HD) is a devastating neurodegenerative disorder caused by a polyglutamine (polyQ) expansion in exon 1 of the Huntingtin (HTT) gene. We have previously demonstrated that spliceosome-mediated trans-splicing is a viable molecular strategy to specifically reduce and repair mutant HTT (mtHTT). Here, the targeted tethering efficacy of the pre-mRNA trans-splicing modules (PTM) in HTT was optimized. Various PTMs that targeted the 3' end of HTT intron 1 or the intron 1 branch point were shown trans-splice into an HTT mini-gene, as well as the endogenous HTT pre-mRNA. PTMs that specifically target the endogenous intron 1 branch point increased the trans-splicing efficacy from 1-5 to 10-15%. Furthermore, lentiviral expression of PTMs in a human HD patient iPSC-derived neural culture significantly reversed two previously established polyQ-length dependent phenotypes. These results suggest that pre-mRNA repair of mtHTT could hold therapeutic benefit and it demonstrates an alternative platform to correct the mRNA product produced by the mtHTT allele in the context of HD. |
X Demographics
The data shown below were collected from the profile of 1 X user who shared this research output. Click here to find out more about how the information was compiled.
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| Switzerland | 1 | 100% |
Demographic breakdown
| Type | Count | As % |
|---|---|---|
| Members of the public | 1 | 100% |
Mendeley readers
The data shown below were compiled from readership statistics for 31 Mendeley readers of this research output. Click here to see the associated Mendeley record.
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| Unknown | 31 | 100% |
Demographic breakdown
| Readers by professional status | Count | As % |
|---|---|---|
| Student > Bachelor | 5 | 16% |
| Student > Ph. D. Student | 4 | 13% |
| Researcher | 4 | 13% |
| Student > Postgraduate | 3 | 10% |
| Student > Doctoral Student | 2 | 6% |
| Other | 6 | 19% |
| Unknown | 7 | 23% |
| Readers by discipline | Count | As % |
|---|---|---|
| Biochemistry, Genetics and Molecular Biology | 7 | 23% |
| Neuroscience | 6 | 19% |
| Agricultural and Biological Sciences | 3 | 10% |
| Engineering | 2 | 6% |
| Medicine and Dentistry | 2 | 6% |
| Other | 4 | 13% |
| Unknown | 7 | 23% |
Attention Score in Context
This research output has an Altmetric Attention Score of 1. 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 20 October 2017.
All research outputs
#22,764,772
of 25,382,440 outputs
Outputs from Frontiers in Neuroscience
#10,138
of 11,542 outputs
Outputs of similar age
#293,008
of 333,631 outputs
Outputs of similar age from Frontiers in Neuroscience
#172
of 180 outputs
Altmetric has tracked 25,382,440 research outputs across all sources so far. This one is in the 1st percentile – i.e., 1% of other outputs scored the same or lower than it.
So far Altmetric has tracked 11,542 research outputs from this source. They typically receive a lot more attention than average, with a mean Attention Score of 11.0. This one is in the 1st percentile – i.e., 1% of its peers scored the same or lower than it.
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We're also able to compare this research output to 180 others from the same source and published within six weeks on either side of this one. This one is in the 1st percentile – i.e., 1% of its contemporaries scored the same or lower than it.