| Title |
A pilot study of direct delivery of hydroxypropyl-beta-cyclodextrin to the lung by the nasal route in a mouse model of Niemann-Pick C1 disease: motor performance is unaltered and lung disease is worsened
|
|---|---|
| Published in |
Journal of Applied Genetics, February 2018
|
| DOI | 10.1007/s13353-018-0431-z |
| Pubmed ID | |
| Authors |
Robert P. Erickson, Gail Deutsch, Ruturaj Patil |
| Abstract |
We have tested the efficacy of hydroxypropyl-beta-cyclodextrin (HPBCD) delivered by the nasal route in the mouse model of juvenile Niemann-Pick C1 disease (NPC1), as pulmonary disease has not responded to systemic therapy with this drug. Since mice have no gag reflex, coating of the nasal cavity, with possible access to the brain, would be followed by delivery of HPBCD to the lung. While foamy macrophages, containing stored cholesterol, were found in the Npc1 nmf164 homozygous mice, a marked inflammatory response was found with inhaled HPBCD, both in mutant and wild-type animals. Slight inflammation also occasionally occurred with saline inhalation. There was no difference between the saline-treated, HPBCD-treated, and untreated Npc1 nmf164 homozygous mice for weight, balance beam performance, or coat hanger performance. Interestingly, there was a trend to longer survival in the HPBCD-treated Npc1 nmf164 homozygous mice, which, when combined with the survival times of the saline-treated survivals (each of which was not different), became significant. |
X Demographics
The data shown below were collected from the profiles of 5 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 | 40% |
| Indonesia | 1 | 20% |
| Unknown | 2 | 40% |
Demographic breakdown
| Type | Count | As % |
|---|---|---|
| Members of the public | 3 | 60% |
| Scientists | 2 | 40% |
Mendeley readers
The data shown below were compiled from readership statistics for 16 Mendeley readers of this research output. Click here to see the associated Mendeley record.
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| Unknown | 16 | 100% |
Demographic breakdown
| Readers by professional status | Count | As % |
|---|---|---|
| Researcher | 3 | 19% |
| Other | 2 | 13% |
| Student > Ph. D. Student | 2 | 13% |
| Student > Master | 2 | 13% |
| Student > Doctoral Student | 1 | 6% |
| Other | 0 | 0% |
| Unknown | 6 | 38% |
| Readers by discipline | Count | As % |
|---|---|---|
| Biochemistry, Genetics and Molecular Biology | 3 | 19% |
| Pharmacology, Toxicology and Pharmaceutical Science | 1 | 6% |
| Linguistics | 1 | 6% |
| Nursing and Health Professions | 1 | 6% |
| Agricultural and Biological Sciences | 1 | 6% |
| Other | 0 | 0% |
| Unknown | 9 | 56% |
Attention Score in Context
This research output has an Altmetric Attention Score of 8. 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 25 November 2021.
All research outputs
#4,198,989
of 23,020,670 outputs
Outputs from Journal of Applied Genetics
#31
of 395 outputs
Outputs of similar age
#94,251
of 437,329 outputs
Outputs of similar age from Journal of Applied Genetics
#2
of 12 outputs
Altmetric has tracked 23,020,670 research outputs across all sources so far. Compared to these this one has done well and is in the 81st percentile: it's in the top 25% of all research outputs ever tracked by Altmetric.
So far Altmetric has tracked 395 research outputs from this source. They receive a mean Attention Score of 3.0. This one has done particularly well, scoring higher than 92% of its peers.
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We're also able to compare this research output to 12 others from the same source and published within six weeks on either side of this one. This one has done well, scoring higher than 83% of its contemporaries.