Evidence map›Paper›PMID 31101939›Full record

ReviewCellular and molecular life sciences : CMLS2019

Targeted enzyme delivery systems in lysosomal disorders: an innovative form of therapy for mucopolysaccharidosis.

Azam Safary, Mostafa Akbarzadeh Khiavi, Yadollah Omidi, Mohammad A Rafi

Abstract readReview
In one paragraph

Review in Cellular and molecular life sciences : CMLS, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.

0numbers the graph read from it
0cells of the map it votes in
11citing papers in PubMed
–field-weighted citation impact
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

11 citing papers in PubMed.

  1. Article
  2. The Role of Visual Electrophysiology in Systemic Hereditary Syndromes.International journal of molecular sciences · 2025
    Review
  3. Article
  4. Bringing enzymes to the proximity party.RSC chemical biology · 2023
    Review
  5. Review
  6. Review
  7. Review
  8. Review
  9. Article
  10. Article
  11. 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

4 authors.

Azam SafaryConnective Tissue Diseases Research Center, Tabriz University of Medical Sciences, Tabriz, Iran.
Mostafa Akbarzadeh KhiaviResearch Center for Pharmaceutical Nanotechnology, Biomedicine Institute, Tabriz University of Medical Sciences, Tabriz, 51656-65811, Iran.
Yadollah OmidiResearch Center for Pharmaceutical Nanotechnology, Biomedicine Institute, Tabriz University of Medical Sciences, Tabriz, 51656-65811, Iran. yomidi@tbzmed.ac.ir.ORCID http://orcid.org/0000-0003-0067-2475
Mohammad A RafiDepartment of Neurology, Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia, PA, 19107, USA. mohammad.rafi@jefferson.edu.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Mucopolysaccharidoses (MPSs), which are inherited lysosomal storage disorders caused by the accumulation of undegraded glycosaminoglycans, can affect the central nervous system (CNS) and elicit cognitive and behavioral issues. Currently used enzyme replacement therapy methodologies often fail to adequately treat the manifestations of the disease in the CNS and other organs such as bone, cartilage, cornea, and heart. Targeted enzyme delivery systems (EDSs) can efficiently cross biological barriers such as blood-brain barrier and provide maximal therapeutic effects with minimal side effects, and hence, offer great clinical benefits over the currently used conventional enzyme replacement therapies. In this review, we provide comprehensive insights into MPSs and explore the clinical impacts of multimodal targeted EDSs.

Indexed as

Enzyme Replacement TherapyBlood-Brain BarrierCell Adhesion MoleculesDrug CarriersGlucosylceramidaseHumansLysosomal Storage DiseasesMucopolysaccharidosesRecombinant ProteinsCell Adhesion MoleculesDrug CarriersGlucosylceramidaseRecombinant ProteinsBlood–brain barrierEnzyme replacement therapyMucopolysaccharidosesNanosystemsTargeted enzyme delivery systems

Identifiers

PMID31101939
PMCPMC11105648

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.