Evidence map›Paper›PMID 36034772›Full record

ArticleMolecular therapy. Methods & clinical development2022

Transduction characteristics of alternative adeno-associated virus serotypes in the cat brain by intracisternal delivery.

Jacqueline E Hunter, Caitlyn M Molony, Jessica H Bagel, Patricia A O'Donnell, Stephen G Kaler, John H Wolfe

Open access · goldAbstract read
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed
1.1field-weighted citation impact, top 20% of its field
1 · What the graph read from 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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

4 citing papers in PubMed, 6 citations in OpenAlex.

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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

6 authors at 3 institutions in 1 country.

Jacqueline E HunterResearch Institute of Children's Hospital of Philadelphia, 502-G Abramson Research Center, 3615 Civic Center Boulevard, Philadelphia, PA 19104, USA.
Caitlyn M MolonyW.F. Goodman Center for Comparative Medical Genetics, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Jessica H BagelW.F. Goodman Center for Comparative Medical Genetics, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Patricia A O'DonnellW.F. Goodman Center for Comparative Medical Genetics, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Stephen G KalerSection on Translational Neuroscience, Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD 20892, USA.
John H WolfeResearch Institute of Children's Hospital of Philadelphia, 502-G Abramson Research Center, 3615 Civic Center Boulevard, Philadelphia, PA 19104, USA.
University of Pennsylvania · USChildren's Hospital of Philadelphia · USEunice Kennedy Shriver National Institute of Child Health and Human Development · US

Funding

Referral Ctr-Animal models of human genetic diseaseP40OD010939 · OD · UNIVERSITY OF PENNSYLVANIA · PI CASAL, MARGRET L · 2012 to 2023
$7.9M
Viral Gene Therapy for Menkes DiseaseU01NS129343 · NINDS · RESEARCH INST NATIONWIDE CHILDREN'S HOSP · PI KALER, STEPHEN GERARD · 2023 to 2025
$3.8M
Translational studies on cerebrospinal fluid (CSF)-directed gene therapy for global neurometabolic brain diseaseR01NS110349 · NINDS · CHILDREN'S HOSP OF PHILADELPHIA · PI WOLFE, JOHN H · 2019 to 2023
$3.3M
Choroid Plexus-Directed Gene Therapy for Alpha-MannosidosisU01HD079066 · NICHD · CHILDREN'S HOSP OF PHILADELPHIA · PI KALER, STEPHEN GERARD, WOLFE, JOHN H · 2015 to 2017
$1.8M
NICHD NIH HHS U01 HD079066NIH HHS P40 OD010939NINDS NIH HHS R01 NS110349NINDS NIH HHS U01 NS129343
6 · The paper itself

Abstract

Multiple studies have examined the transduction characteristics of different AAV serotypes in the mouse brain, where they can exhibit significantly different patterns of transduction. The pattern of transduction also varies with the route of administration. Much less information exists for the transduction characteristics in large-brained animals. Large animal models have brains that are closer in size and organization to the human brain, such as being gyrencephalic compared to the lissencephalic rodent brains, pathway organization, and certain electrophysiologic properties. Large animal models are used as translational intermediates to develop gene therapies to treat human diseases. Various AAV serotypes and routes of delivery have been used to study the correction of pathology in the brain in lysosomal storage diseases. In this study, we evaluated the ability of selected AAV serotypes to transduce cells in the cat brain when delivered into the cerebrospinal fluid via the

Indexed as

AAVcatcisterna magnagyrencephalicintrathecallarge animal brainserotypes

Identifiers

PMID36034772
PMCPMC9391516
OpenAlexW4285605490

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.