ReviewFrontiers in neurology2021
Recombinant Adeno-Associated Virus Serotype 9 Gene Therapy in Spinal Muscular Atrophy.
Review in Frontiers in neurology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 papers.
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
27 citing papers in PubMed, 44 citations in OpenAlex.
- Delphi consensus on gene therapy of spinal muscular atrophy with onasemnogene abeparvovec in Germany, Austria and Switzerland - part II - expert based recommendations for surveillance and management of side-effects.Journal of neuromuscular diseases · 2026Article
- Optimization of CEST MRI Reporter Protein Design Using Cation-Pi Networks.Chemistry (Weinheim an der Bergstrasse, Germany) · 2026Article
- Prevalence of pre-existing neutralizing antibodies to AAV5 and AAV8 in patients with Wilson's disease.Molecular therapy. Advances · 2026Article
- MyoRep: A Novel Reporter System to Detect Early Muscle Atrophy In Vitro and In Vivo.Journal of cachexia, sarcopenia and muscle · 2026Article
- The role of sleep-disordered breathing in adolescents with spinal muscular atrophy.Sleep & breathing = Schlaf & Atmung · 2026Review
- Comparison of AAV9-driven motor neuron transduction following different CNS-directed delivery methods in mice.Scientific reports · 2026Article
- Novel adenovirus vaccine vectors lacking thrombosis-associated interactions with platelet factor 4.iScience · 2026Article
- The amazing AAV capsids: Into the structure-verse.Molecular therapy. Methods & clinical development · 2025Review
- Proprioceptive synaptic dysfunction is a key feature in mice and humans with spinal muscular atrophy.Brain : a journal of neurology · 2025Article
- Dynamic activation of rAAV transgene expression by a small molecule that recruits endogenous transcriptional machinery.Nucleic acids research · 2025Article
- Alterations in cardiac function correlate with a disruption in fatty acid metabolism in a mouse model of SMA.Human molecular genetics · 2025Article
- Catecholaminergic dysfunction drives postural and locomotor deficits in a mouse model of spinal muscular atrophy.Cell reports · 2025Article
- Article
- Sleeping Beauty mRNA-LNP enables stable rAAV transgene expression in mouse and NHP hepatocytes and improves vector potency.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- Article
- Gene and cell therapy of human genetic diseases: Recent advances and future directions.Journal of cellular and molecular medicine · 2024Review
- Abortive and productive infection of CNS cell types following in vivo delivery of VSV.Proceedings of the National Academy of Sciences of the United States of America · 2024Article
- Therapeutic strategy for spinal muscular atrophy by combining gene supplementation and genome editing.Nature communications · 2024Article
- Onasemnogene Abeparvovec Administration via Peripherally Inserted Central Catheter: A Case Report.Children (Basel, Switzerland) · 2024Article
- Glial-restricted progenitor cells: a cure for diseased brain?Biological research · 2024Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 3 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease caused by deletion or mutation of the
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.