Evidence map›Paper›PMID 28660346›Full record

ReviewJournal of molecular medicine (Berlin, Germany)2017

Adeno-associated viral gene therapy for mucopolysaccharidoses exhibiting neurodegeneration.

Adeline A Lau, Kim M Hemsley

Abstract readReview
PubMed Publisher
In one paragraph

Review in Journal of molecular medicine (Berlin, Germany), 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.

0numbers the graph read from it
0cells of the map it votes in
5citing papers in PubMed
0.9field-weighted citation impact, top 25% 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

5 citing papers in PubMed, 8 citations in OpenAlex.

  1. Convection-Enhanced Delivery in Children: Techniques and Applications.Advances and technical standards in neurosurgery · 2022
    Review
  2. Article
  3. Article
  4. Review
  5. Review
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

2 authors at 1 institution in 1 country.

Adeline A LauLysosomal Diseases Research Unit, Nutrition and Metabolism Theme, South Australian Health and Medical Research Institute (SAHMRI), PO Box 11060, Adelaide, South Australia, 5001, Australia. adeline.lau@sahmri.com.
Kim M HemsleyLysosomal Diseases Research Unit, Nutrition and Metabolism Theme, South Australian Health and Medical Research Institute (SAHMRI), PO Box 11060, Adelaide, South Australia, 5001, Australia.
South Australian Health and Medical Research Institute · AU

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The mucopolysaccharidoses (MPS) are a subgroup of lysosomal storage disorders that are caused by mutations in the genes involved in glycosaminoglycan breakdown. Multiple organs and tissues are affected, including the central nervous system. At present, hematopoietic stem cell transplantation and enzyme replacement therapies are approved for some of the (non-neurological) MPS. Treatments that effectively ameliorate the neurological aspects of the disease are being assessed in clinical trials. This review will focus on the recent outcomes and planned viral vector-mediated gene therapy clinical trials, and the pre-clinical data that supported these studies, for MPS-I (Hurler/Scheie syndrome), MPS-II (Hunter syndrome), and MPS-IIIA and -IIIB (Sanfilippo syndrome).

Indexed as

AnimalsClinical Trials as TopicDependovirusEnzyme Replacement TherapyGenetic TherapyGenetic VectorsGlycosaminoglycansHumansLysosomesMucopolysaccharidosesMutationGlycosaminoglycansCentral nervous systemGene therapyLysosomal storage diseaseMucopolysaccharidosisViral vectors

Identifiers

PMID28660346
OpenAlexW2730299814

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.