ReviewNature reviews. Neuroscience2024
Gene therapy for CNS disorders: modalities, delivery and translational challenges.
Review in Nature reviews. Neuroscience, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 52 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
52 citing papers in PubMed.
- Intranasal versus intravenous AAV delivery: A comparative analysis of brain-targeting efficiency and peripheral exposure in mice.Gene therapy · 2026Article
- Dual-target gene therapy in Parkinson's disease: a multicenter phase 1 trial.Nature medicine · 2026Article
- Attenuating AAV-triggered innate immunity in the adult mouse nervous systemActa pharmaceutica Sinica. B · 2026Article
- Noninvasive Focal Gene Delivery into the Cerebellum of Non-Human Primates using Focused Ultrasound.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- NeuroD1 gene therapy inhibits glioma growth and extends life span throughMolecular therapy. Oncology · 2026Article
- Swine reporter model for preclinical evaluation and characterization of gene delivery vectors.Molecular therapy. Advances · 2026Article
- Prime editing in neuropsychiatric disorders: From mutation-specific target selection to clinical translation.Neuroprotection (Chichester, England) · 2026Review
- Ionizable Polymeric Micelles Targeting Transferrin Receptor 1 Enhance Systemic mRNA Delivery to the Brain.Molecular pharmaceutics · 2026Article
- Metal-Supramolecular Drug Delivery System Empowered Meningeal Lymphatic Vessels-Bridged Intracranial-Peripheral Dual Immune Modulation for Reversing Glioblastoma Immune Suppression.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- Recent Advances in the Non-viral Delivery of Genes to Central Nervous System Disorders.Cellular and molecular neurobiology · 2026Review
- Design and initial characterization of a novel mini-promoter for gene therapies targeting the central nervous system.Molecular therapy. Advances · 2026Article
- Global Research Trends and Hotspots in Gene Editing and Stem Cell Therapies for Neurodegenerative Diseases: Bibliometric and Visualization Analysis.Interactive journal of medical research · 2026Review
- Gene therapy for epilepsy: An emerging, promising approach for a serious neurological disorder.Journal of internal medicine · 2026Review
- Article
- A disease-severity-responsive nanoparticle enables potent ghrelin messenger RNA therapy in osteoarthritis.Nature nanotechnology · 2026Article
- Engineering a human-based translational activator for targeted protein expression restoration.Nucleic acids research · 2026Article
- Hereditary spastic paraplegia: from decades of therapy to future innovations.Therapeutic advances in neurological disorders · 2026Review
- Analysis and comparison of the trends in the burden of motor neuron disease in China and worldwide from 1990 to 2021.PloS one · 2026Article
- Production and Purification of Lentiviral Vectors.Methods in molecular biology (Clifton, N.J.) · 2026Article
- AAV-mediated gene therapy for Alzheimer's disease: neuroprotective mechanisms and translational challenges.Frontiers in aging neuroscience · 2026Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
14 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Gene therapy is emerging as a powerful tool to modulate abnormal gene expression, a hallmark of most CNS disorders. The transformative potentials of recently approved gene therapies for the treatment of spinal muscular atrophy (SMA), amyotrophic lateral sclerosis (ALS) and active cerebral adrenoleukodystrophy are encouraging further development of this approach. However, most attempts to translate gene therapy to the clinic have failed to make it to market. There is an urgent need not only to tailor the genes that are targeted to the pathology of interest but to also address delivery challenges and thereby maximize the utility of genetic tools. In this Review, we provide an overview of gene therapy modalities for CNS diseases, emphasizing the interconnectedness of different delivery strategies and routes of administration. Important gaps in understanding that could accelerate the clinical translatability of CNS genetic interventions are addressed, and we present lessons learned from failed clinical trials that may guide the future development of gene therapies for the treatment and management of CNS disorders.
Indexed as
Identifiers
38898231What 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.