Evidence map›Paper›PMID 36398557›Full record

ReviewZhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics2022

[Application of adeno-associated virus-mediated gene therapy in lysosomal storage diseases].

Xue-Qin Lin, Xiao-Le Wang, Jing Peng

Abstract readReviewEnglish Abstract
PubMed Full text at PMC Publisher
In one paragraph

Review in Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. 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

3 authors.

Xue-Qin LinDepartment of Pediatrics, Xiangya Hospital, Central South University/Clinical Rearch Center for Children's Neurodevelopmental Disabilities of Hunan Province, Changsha 410008, China.
Xiao-Le WangDepartment of Pediatrics, Xiangya Hospital, Central South University/Clinical Rearch Center for Children's Neurodevelopmental Disabilities of Hunan Province, Changsha 410008, China.
Jing PengDepartment of Pediatrics, Xiangya Hospital, Central South University/Clinical Rearch Center for Children's Neurodevelopmental Disabilities of Hunan Province, Changsha 410008, China.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Lysosomal storage disorders (LSDs) are a group of single-gene inherited metabolic diseases caused by defects in lysosomal enzymes or function-related proteins. Enzyme replacement therapy is the main treatment method in clinical practice, but it has a poor effect in patients with neurological symptoms. With the rapid development of multi-omics, sequencing technology, and bioengineering, gene therapy has been applied in patients with LSDs. As one of the vectors of gene therapy, adeno-associated virus (AAV) has good prospects in the treatment of genetic and metabolic diseases. More and more studies have shown that AAV-mediated gene therapy is effective in LSDs. This article reviews the application of AAV-mediated gene therapy in LSDs.

Indexed as

DependovirusLysosomal Storage DiseasesEnzyme Replacement TherapyGenetic TherapyHumansProteinsProteinsAdeno-associated virusGene therapyLysosomal storage disease

Identifiers

PMID36398557
PMCPMC9678058

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.