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Using antisense technology to develop a novel therapy for α-1 antitrypsin deficient (AATD) liver disease and to model AATD lung disease

Overview of attention for article published in Rare Diseases, March 2014
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Title
Using antisense technology to develop a novel therapy for α-1 antitrypsin deficient (AATD) liver disease and to model AATD lung disease
Published in
Rare Diseases, March 2014
DOI 10.4161/rdis.28511
Pubmed ID
Authors

Shuling Guo, Sheri L Booten, Andrew Watt, Luis Alvarado, Susan M Freier, Jeffery H Teckman, Michael L McCaleb, Brett P Monia

Abstract

Alpha-1 antitrypsin (AAT) is a serum protease inhibitor that belongs to the serpin superfamily. Mutations in AAT are associated with α-1 antitrypsin deficiency (AATD), a rare genetic disease with two distinct manifestations: AATD lung disease and AATD liver disease. AATD lung disease is caused by loss-of-function of AAT and can be treated with plasma-derived AAT. AATD liver disease is due to the aggregation and retention of mutant AAT protein in the liver; the only treatment available for AATD liver disease is liver transplantation. Here we demonstrate that antisense oligonucleotides (ASOs) targeting human AAT efficiently reduce levels of both short and long human AAT transcript in vitro and in transgenic mice, providing a novel therapy for AATD liver disease. In addition, ASO-mediated depletion of mouse AAT may offer a useful animal model for the investigation of AATD lung disease.

Mendeley readers

Mendeley readers

The data shown below were compiled from readership statistics for 32 Mendeley readers of this research output. Click here to see the associated Mendeley record.

Geographical breakdown

Country Count As %
United States 1 3%
Unknown 31 97%

Demographic breakdown

Readers by professional status Count As %
Researcher 11 34%
Other 5 16%
Student > Bachelor 3 9%
Student > Ph. D. Student 3 9%
Student > Doctoral Student 2 6%
Other 3 9%
Unknown 5 16%
Readers by discipline Count As %
Biochemistry, Genetics and Molecular Biology 10 31%
Agricultural and Biological Sciences 8 25%
Chemistry 3 9%
Pharmacology, Toxicology and Pharmaceutical Science 2 6%
Medicine and Dentistry 2 6%
Other 1 3%
Unknown 6 19%
Attention Score in Context

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 28 November 2014.
All research outputs
#22,760,732
of 25,377,790 outputs
Outputs from Rare Diseases
#43
of 46 outputs
Outputs of similar age
#205,698
of 235,689 outputs
Outputs of similar age from Rare Diseases
#3
of 3 outputs
Altmetric has tracked 25,377,790 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 46 research outputs from this source. They typically receive a lot more attention than average, with a mean Attention Score of 17.7. This one scored the same or higher as 3 of them.
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 235,689 tracked outputs that were published within six weeks on either side of this one in any source. This one is in the 1st percentile – i.e., 1% of its contemporaries scored the same or lower than it.
We're also able to compare this research output to 3 others from the same source and published within six weeks on either side of this one.