Trial reportScience translational medicine2020
Slowing late infantile Batten disease by direct brain parenchymal administration of a rh.10 adeno-associated virus expressing
Open access · greenAbstract readClinical Trial
In one paragraph
Trial report in Science translational medicine, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 48 papers, 2 of them syntheses that pooled it.
0numbers the graph read from it
0cells of the map it votes in
48citing papers in PubMed, 2 pooled it
4.5field-weighted citation impact, top 4% of its field
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
48 citing papers in PubMed, 2 syntheses or guidelines pooled it, 69 citations in OpenAlex.
- rAAV immunogenicity, toxicity, and durability in 255 clinical trials: A meta-analysis.Frontiers in immunology · 2022Pooled it
- Gene Therapy Advances: A Meta-Analysis of AAV Usage in Clinical Settings.Frontiers in medicine · 2021Pooled it
- Consequences of human genetic variations inMolecular therapy. Advances · 2026Article
- Optimization of systemic AAV9 gene therapy in Niemann-Pick disease, type C1 mice.Life science alliance · 2026Article
- Caregiver Reports of Neurodevelopmental Functions in Pediatric Lysosomal Storage Disorders: A Scoping Review.Journal of inherited metabolic disease · 2026Article
- Genetic therapies for neurological diseases.Pharmacological reviews · 2026Review
- Roadmap for direct and indirect translation of optogenetics into discoveries and therapies for humans.Nature neuroscience · 2025Review
- MR-Guidance of Gene Therapy for Brain Diseases: Moving From Palliative Treatment to Cures.Journal of magnetic resonance imaging : JMRI · 2025Review
- Gene editing for Spinocerebellar ataxia type 3 taking advantage of the human ATXN3L paralog as replacement gene.Gene therapy · 2025Article
- Autosomal Recessive Cerebellar Ataxias: Translating Genes to Therapies.Annals of neurology · 2025Review
- Current clinical applications of AAV-mediated gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- AAV vectors trigger DNA damage response-dependent pro-inflammatory signalling in human iPSC-derived CNS models and mouse brain.Nature communications · 2025Article
- Drug delivery pathways to the central nervous system via the brain glymphatic system circumventing the blood-brain barrier.Exploration (Beijing, China) · 2025Review
- Neuronal Ceroid Lipofuscinosis-Concepts, Classification, and Avenues for Therapy.CNS neuroscience & therapeutics · 2025Review
- Adeno-associated virus vectors and neurotoxicity-lessons from preclinical and human studies.Gene therapy · 2025Review
- Viral and non-viral cellular therapies for neurodegeneration.Frontiers in medicine · 2025Review
- Advances in AAV-mediated gene replacement therapy for pediatric monogenic neurological disorders.Molecular therapy. Methods & clinical development · 2024Review
- Next-generation strategies to improve safety and efficacy of adeno-associated virus-based gene therapy for hemophilia: lessons from clinical trials in other gene therapies.Haematologica · 2024Review
- Neuroimaging Applications for the Delivery and Monitoring of Gene Therapy for Central Nervous System Diseases.Human gene therapy · 2024Review
- Different diseases, different needs: Patient preferences for gene therapy in lysosomal storage disorders, a probabilistic threshold technique survey.Orphanet journal of rare diseases · 2024Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
24 authors at 1 institution in 1 country.
Dolan SondhiDepartment of Genetic Medicine, Weill Cornell Medical College, New York, NY 10065, USA.ORCID 0000-0002-2218-5910
Stephen M KaminskyDepartment of Genetic Medicine, Weill Cornell Medical College, New York, NY 10065, USA.
Neil R HackettDepartment of Genetic Medicine, Weill Cornell Medical College, New York, NY 10065, USA.
Odelya E PagovichDepartment of Genetic Medicine, Weill Cornell Medical College, New York, NY 10065, USA.
Jonathan B RosenbergDepartment of Genetic Medicine, Weill Cornell Medical College, New York, NY 10065, USA.
Bishnu P DeDepartment of Genetic Medicine, Weill Cornell Medical College, New York, NY 10065, USA.
Alvin ChenDepartment of Genetic Medicine, Weill Cornell Medical College, New York, NY 10065, USA.
Benjamin Van de GraafDepartment of Genetic Medicine, Weill Cornell Medical College, New York, NY 10065, USA.
Jason G MezeyDepartment of Genetic Medicine, Weill Cornell Medical College, New York, NY 10065, USA.
Grace W MammenDepartment of Genetic Medicine, Weill Cornell Medical College, New York, NY 10065, USA.
Denesy MancenidoDepartment of Genetic Medicine, Weill Cornell Medical College, New York, NY 10065, USA.
Fang XuDepartment of Genetic Medicine, Weill Cornell Medical College, New York, NY 10065, USA.
Barry KosofskyDepartment of Pediatrics, Weill Cornell Medical College, New York, NY 10065, USA.
Kaleb YohayDepartment of Pediatrics, Weill Cornell Medical College, New York, NY 10065, USA.ORCID 0000-0001-5560-3392
Stefan WorgallDepartment of Genetic Medicine, Weill Cornell Medical College, New York, NY 10065, USA.ORCID 0000-0003-3716-2890
Robert J KanerDepartment of Genetic Medicine, Weill Cornell Medical College, New York, NY 10065, USA.ORCID 0000-0003-0527-576X
Mark SouwedaineDepartment of Neurological Surgery, Weill Cornell Medical College, New York, NY 10065, USA.ORCID 0000-0001-5627-4551
Bruce M GreenwaldDepartment of Pediatrics, Weill Cornell Medical College, New York, NY 10065, USA.
Michael KaplittDepartment of Neurological Surgery, Weill Cornell Medical College, New York, NY 10065, USA.ORCID 0000-0002-8979-2459
Jonathan P DykeDepartment of Radiology, Weill Cornell Medical College, New York, NY 10065, USA.ORCID 0000-0001-7170-488X
Douglas J BallonDepartment of Genetic Medicine, Weill Cornell Medical College, New York, NY 10065, USA.ORCID 0000-0002-7162-9020
Linda A HeierDepartment of Radiology, Weill Cornell Medical College, New York, NY 10065, USA.ORCID 0000-0001-5519-9326
Szilard KissDepartment of Ophthalmology, Retina Service, Weill Cornell Medical College, New York, NY 10065, USA.ORCID 0000-0003-3433-8432
Ronald G CrystalDepartment of Genetic Medicine, Weill Cornell Medical College, New York, NY 10065, USA. geneticmedicine@med.cornell.edu.ORCID 0000-0001-6177-6839
Cornell University · US
Funding
Disparities in COVID Disease Severity and Outcomes in New York CityUL1TR002384 · NCATS · WEILL MEDICAL COLL OF CORNELL UNIV · PI JULIANNE L IMPERATO-MCGINLEY · 2017 to 2026
$86.2MClinical and Translational Science CenterUL1TR000457 · NCATS · WEILL MEDICAL COLL OF CORNELL UNIV · PI IMPERATO-MCGINLEY, JULIANNE L · 2012 to 2016
$46.0MTraining & EducationU54NS065768 · NINDS · UNIVERSITY OF MINNESOTA · PI WHITLEY, CHESTER B. · 2009 to 2024
$17.7MGene Therapy for Batten Disease Using AAVrh.10 Expressing Human CLN2 cDNAR01NS061848 · NINDS · WEILL MEDICAL COLL OF CORNELL UNIV · PI CRYSTAL, RONALD G · 2010 to 2013
$3.7MNCATS NIH HHS UL1 TR000457NCATS NIH HHS UL1 TR002384NINDS NIH HHS R01 NS061848NINDS NIH HHS U54 NS065768
Abstract
Late infantile Batten disease (CLN2 disease) is an autosomal recessive, neurodegenerative lysosomal storage disease caused by mutations in the
Indexed as
DependovirusNeuronal Ceroid-LipofuscinosesAminopeptidasesBrainChildDipeptidyl-Peptidases and Tripeptidyl-PeptidasesGenetic TherapyHumansMagnetic Resonance ImagingTripeptidyl-Peptidase 1AminopeptidasesDipeptidyl-Peptidases and Tripeptidyl-PeptidasesTPP1 protein, humanTripeptidyl-Peptidase 1
Identifiers
What OpenQuestion holds
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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.