Evidence map›Paper›PMID 25208876›Full record

ArticlePharmaceutical research2015

Laronidase-functionalized multiple-wall lipid-core nanocapsules: promising formulation for a more effective treatment of mucopolysaccharidosis type I.

Fabiana Quoos Mayer, Márcia Duarte Adorne, Eduardo André Bender, Talita Giacomet de Carvalho, Anna Cláudia Dilda, Ruy Carlos Ruver Beck, Sílvia Stanisçuaski Guterres, Roberto Giugliani, Ursula Matte, Adriana Raffin Pohlmann

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Article in Pharmaceutical research, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.

0numbers the graph read from it
0cells of the map it votes in
15citing papers in PubMed
1.9field-weighted citation impact, top 15% 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

15 citing papers in PubMed, 40 citations in OpenAlex.

  1. Article
  2. Review
  3. Article
  4. Review
  5. Article
  6. Article
  7. Article
  8. Mucopolysaccharidosis IVA: Diagnosis, Treatment, and Management.International journal of molecular sciences · 2020
    Review
  9. Article
  10. Review
  11. Possible strategies to cross the blood-brain barrier.Italian journal of pediatrics · 2018
    Review
  12. Article
  13. Article
  14. Article
  15. Article
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

10 authors at 3 institutions in 1 country.

Fabiana Quoos MayerGene Therapy Center, Experimental Research Center, Hospital de Clínicas de Porto Alegre, Porto Alegre, RS, Brazil.
Márcia Duarte Adorne
Eduardo André Bender
Talita Giacomet de Carvalho
Anna Cláudia Dilda
Ruy Carlos Ruver Beck
Sílvia Stanisçuaski Guterres
Roberto Giugliani
Ursula Matte
Adriana Raffin Pohlmann
Universidade Federal do Rio Grande do Sul · BRHospital de Clínicas de Porto Alegre · BRNational Institute on Population Medical Genetics · BR

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

purposeMucopolysaccharidosis I is a genetic disorder caused by alpha-L-iduronidase deficiency. Its primary treatment is enzyme replacement therapy (ERT), which has limitations such as a high cost and a need for repeated infusions over the patient's lifetime. Considering that nanotechnological approaches may enhance enzyme delivery to organs and can reduce the dosage thereby enhancing ERT efficiency and/or reducing its cost, we synthesized laronidase surface-functionalized lipid-core nanocapsules (L-MLNC).

methodsL-MLNCs were synthesized by using a metal complex. Size distributions were evaluated by laser diffraction and dynamic light scattering. The kinetic properties, cytotoxicity, cell uptake mechanisms, clearance profile and biodistribution were evaluated.

resultsSize distributions showed a D[4,3] of 134 nm and a z-average diameter of 71 nm. L-MLNC enhanced the Vmax and Kcat in comparison with laronidase. L-MLNC is not cytotoxic, and nanocapsule uptake by active transport is not only mediated by mannose-6-phosphate receptors. The clearance profile is better for L-MLNC than for laronidase. A biodistribution analysis showed enhanced enzyme activity in different organs within 4 h and 24 h for L-MLNC.

conclusionsThe use of lipid-core nanocapsules as building blocks to synthesize surface-functionalized nanocapsules represents a new platform for producing decorated soft nanoparticles that are able to modify drug biodistribution.

Indexed as

Enzyme Replacement TherapyNanocapsulesAnimalsArea Under CurveBiological TransportCells, CulturedCell SurvivalChemistry, PharmaceuticalFibroblastsIduronidaseInjections, IntravenousLipidsMetabolic Clearance RateMice, KnockoutMucopolysaccharidosis INanomedicineIduronidaseLipidsNanocapsules

Identifiers

PMID25208876
OpenAlexW1994710775

What OpenQuestion holds

Textmetadata
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.