ReviewNeuron2019
Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality.
Review in Neuron, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 227 papers, 1 of them a synthesis that pooled 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.
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
227 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Lentivirus- or AAV-mediated gene therapy interventions in ischemic stroke: A systematic review of preclinicalJournal of cerebral blood flow and metabolism : official journal of the International Society of Cerebral Blood Flow and Metabolism · 2022Pooled it
- Slowing late infantile Batten disease by direct brain parenchymal administration of a rh.10 adeno-associated virus expressingScience translational medicine · 2020Trial
- Controllable gene delivery via masked adeno-associated viral vectors.Nature materials · 2026Article
- Article
- Whole-brain biodistribution of AAV2, AAV8, and AAV-B10 after unilateral intracerebroventricular injection.Molecular therapy. Advances · 2026Article
- Gene delivery to the mouse brain: Facilitating disease modeling and gene therapy.Neural regeneration research · 2026Article
- Crosstalk-free optical indicator-actuator combinations for bidirectional optogenetics.npj biomedical innovations · 2026Review
- RNA-based therapeutics for Alzheimer's disease and related tauopathies: challenges and opportunities.The journal of prevention of Alzheimer's disease · 2026Review
- Autonomous intranasal delivery systems for central nervous system therapeutics.Experimental & molecular medicine · 2026Review
- CRISPR-Cas9-based therapies for Huntington's disease and Friedreich's ataxia: mechanisms, advances, and future perspectives.Neurogenetics · 2026Review
- Dual-target gene therapy in Parkinson's disease: a multicenter phase 1 trial.Nature medicine · 2026Article
- Comparative Transduction Profiling of Four Intravenously Delivered AAV Capsids in the Neonatal Murine Nervous System.Biomedicines · 2026Article
- Para-uterine imaging protocol: Stabilizing living mouse embryos within the maternal abdomen for in vivo imaging and visually guided physiology.STAR protocols · 2026Article
- AAV immuno-gene therapy platform delivering vectorized cytokines defines a new modality for high-grade glioma treatment.Molecular therapy. Oncology · 2026Article
- Adeno-associated virus-induced neurotoxicity is prevented by CpG depletion.Molecular therapy. Advances · 2026Article
- Multi-functionalized chitosan-Extracellular vesicles nanohybrid system for intranasal delivery of pApoE2 to attenuate age-related inflammation.International journal of biological macromolecules · 2026Article
- MyoRep: A Novel Reporter System to Detect Early Muscle Atrophy In Vitro and In Vivo.Journal of cachexia, sarcopenia and muscle · 2026Article
- Defining a Midgestational Window forbioRxiv : the preprint server for biology · 2026Article
- Extracellular Vesicles in Alzheimer's Disease: Mechanisms, Biomarkers, and Therapeutic Engineering.International journal of molecular sciences · 2026Review
- Review
167 more citing papers are in PubMed but not listed here.
Corrections and comments
- Erratum issued
Authors and funding
2 authors.
Funding
Abstract
Gene transfer has long been a compelling yet elusive therapeutic modality. First mainly considered for rare inherited disorders, gene therapy may open treatment opportunities for more challenging and complex diseases such as Alzheimer's or Parkinson's disease. Today, examples of striking clinical proof of concept, the first gene therapy drugs coming onto the market, and the emergence of powerful new molecular tools have broadened the number of avenues to target neurological disorders but have also highlighted safety concerns and technology gaps. The vector of choice for many nervous system targets currently is the adeno-associated viral (AAV) vector due to its desirable safety profile and strong neuronal tropism. In aggregate, the clinical success, the preclinical potential, and the technological innovation have made therapeutic AAV drug development a reality, particularly for nervous system disorders. Here, we discuss the rationale, opportunities, limitations, and progress in clinical AAV gene therapy.
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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.