Evidence map›Paper›PMID 31666200›Full record

ArticleNanomedicine : nanotechnology, biology, and medicine2020

Enriched chitosan nanoparticles loaded with siRNA are effective in lowering Huntington's disease gene expression following intranasal administration.

Vasyl Sava, Oksana Fihurka, Anastasia Khvorova, Juan Sanchez-Ramos

Open access · greenAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Nanomedicine : nanotechnology, biology, and medicine, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 43 papers.

0numbers the graph read from it
0cells of the map it votes in
43citing papers in PubMed
4.6field-weighted citation impact, top 4% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

43 citing papers in PubMed, 96 citations in OpenAlex.

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  17. Spatiotemporal tracking of gold nanorods after intranasal administration for brain targeting.Journal of controlled release : official journal of the Controlled Release Society · 2023
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

4 authors at 2 institutions in 1 country.

Vasyl SavaDepartment of Neurology, University of South Florida, Tampa, FL.
Oksana FihurkaDepartment of Neurology, University of South Florida, Tampa, FL.
Anastasia KhvorovaRNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, USA.
Juan Sanchez-RamosDepartment of Neurology, University of South Florida, Tampa, FL. Electronic address: jsramos@health.usf.edu.
University of South Florida · USUniversity of Massachusetts Chan Medical School · US

Funding

Nanocarriers Designed to Deliver Nucleic Acids to BrainR01NS095563 · NINDS · UNIVERSITY OF SOUTH FLORIDA · PI SANCHEZ-RAMOS, JUAN R · 2015 to 2019
$2.0M
Mid-Scale RNA Synthesis, Purification and Quality Control SystemS10OD020012 · OD · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI KHVOROVA, ANASTASIA · 2015 to 2015
$563k
NIH HHS S10 OD020012NINDS NIH HHS R01 NS095563
6 · The paper itself

Abstract

Therapies to lower gene expression in brain disease currently require chronic administration into the cerebrospinal fluid (CSF) by intrathecal infusions or direct intracerebral injections. Though well-tolerated in the short-term, this approach is not tenable for a life-time of administration. Nose-to-brain delivery of enriched chitosan-based nanoparticles loaded with anti-HTT siRNA was studied in a transgenic YAC128 mouse model of Huntington's Disease (HD). A series of chitosan-based nanoparticle (NP) formulations encapsulating anti-HTT small interfering RNA (siRNA) was designed to protect the payload from degradation "en route" to the target. Factors to improve production of effective nanocarriers of anti-HTT siRNA were identified and tested in a YAC128 mouse model of Huntington's disease. Four formulations of nanocarriers were identified to be effective in lowering HTT mRNA expression by at least 50%. Intranasal administration of nanoparticles carrying siRNA is a promising therapeutic alternative for safe and effective lowering of mutant HTT expression.

Indexed as

ChitosanHuntingtin ProteinHuntington DiseaseNanoparticlesRNA, Small InterferingAdministration, IntranasalAnimalsDisease Models, AnimalGene Expression RegulationMiceMice, TransgenicChitosanHtt protein, mouseHuntingtin ProteinRNA, Small InterferingGene-therapyHuntington's diseaseIntranasal administrationNanocarriersYAC128 mouse model

Identifiers

PMID31666200
PMCPMC7098382
OpenAlexW2982227296

What OpenQuestion holds

Textmetadata
LicenceTDM
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.