ReviewGene therapy2020
Gene therapeutic strategies and relevant clinical trials in neuromuscular disorder in China.
Review in Gene therapy, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
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.
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.
Who cites it
1 citing paper in PubMed, 1 citations in OpenAlex.
- Spotlight on gene therapy in China.Gene therapy · 2020Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Neuromuscular disorder is a diverse group of genetic disease, among which Duchenne muscular dystrophy and Spinal muscular atrophy are most common. Recently, the great breakthroughs of gene targeted therapeutic strategies are leading a profound revolution in the standard care of neuromuscular disorders over the world including China. This review will offer an outline of the molecular pathogenesis, clinical progress, critical trials, as well as the challenges of new gene therapy in the treatment of Spinal muscular atrophy and Duchenne muscular dystrophy in China, mainly includes mRNA splicing modulators and adeno-associated virus mediated gene replacement. We hope to highlight some important findings about the critical development of gene therapy in this field, which might be helpful for suggesting potential therapeutic treatment for neuromuscular disease in China.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
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.