Evidence map›Paper›PMID 14502216›Full record

ArticleGene therapy2003

Adeno-associated virus vector-mediated transduction in the cat brain.

Charles H Vite, Marco A Passini, Mark E Haskins, John H Wolfe

Abstract read
PubMed Publisher
In one paragraph

Article in Gene therapy, 2003. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 50 papers.

0numbers the graph read from it
0cells of the map it votes in
50citing papers in PubMed
4.4field-weighted citation impact, top 5% 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

50 citing papers in PubMed, 111 citations in OpenAlex.

  1. Article
  2. Article
  3. Adeno-Associated Viral Vectors in the Treatment of Epilepsy.International journal of molecular sciences · 2024
    Review
  4. Article
  5. Article
  6. Article
  7. Review
  8. Article
  9. Article
  10. Targeting Age-Related Neurodegenerative Diseases by AAV-Mediated Gene Therapy.Advances in experimental medicine and biology · 2021
    Article
  11. Article
  12. Review
  13. Article
  14. Article
  15. In Situ Hybridization for Detection of AAV-Mediated Gene Expression.Methods in molecular biology (Clifton, N.J.) · 2019
    Article
  16. Review
  17. Review
  18. Toxicology and Biodistribution: The Clinical Value of Animal Biodistribution Studies.Molecular therapy. Methods & clinical development · 2018
    Review
  19. Review
  20. Review
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

4 authors at 1 institution in 1 country.

Charles H ViteSchool of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Marco A Passini
Mark E Haskins
John H Wolfe
University of Pennsylvania · US

Funding

Gene transfer &NMR studies in alpha-mannosidosis brainR01DK063973 · NIDDK · CHILDREN'S HOSP OF PHILADELPHIA · PI WOLFE, JOHN H · 2002 to 2025
$11.0M
Referral Ctr-Animal models of human genetic diseaseP40OD010939 · OD · UNIVERSITY OF PENNSYLVANIA · PI CASAL, MARGRET L · 2012 to 2023
$7.9M
X-LINKED SEVERE COMBINED IMMUNODEFICIENCY IN THE DOGP40RR002512 · NCRR · UNIVERSITY OF PENNSYLVANIA · PI HASKINS, MARK E · 1985 to 2011
$7.6M
Disseminated AAV Vector Transport in the Brain Via Neuronal PathwaysR01NS038690 · NINDS · CHILDREN'S HOSP OF PHILADELPHIA · PI WOLFE, JOHN H · 2000 to 2015
$6.7M
GENE THERAPY TRAINING--CYSTIC FIBROSIS/GENETIC DISEASEST32DK007748 · NIDDK · UNIVERSITY OF PENNSYLVANIA · PI WOLFE, JOHN H · 1997 to 2011
$2.8M
IN UTERO TRANSPLANTATION IN CATSR01DK049652 · NIDDK · UNIVERSITY OF WASHINGTON · PI ABKOWITZ, JANIS L · 1994 to 2002
$807k
MRI, MTI AND MRS AND MPS VII AND KRABBE DISEASEK08NS002032 · NINDS · UNIVERSITY OF PENNSYLVANIA · PI VITE, CHARLES H · 1998 to 2003
$537k
NCRR NIH HHS RR02512NIDDK NIH HHS DK07748NIDDK NIH HHS DK47747NIDDK NIH HHS DK49652NIDDK NIH HHS DK63973NIDDK NIH HHS R01 DK063973NIH HHS P40 OD010939NINDS NIH HHS K08 NS02032NINDS NIH HHS NS38690NINDS NIH HHS R01 NS038690
6 · The paper itself

Abstract

Adeno-associated virus (AAV) vectors are capable of delivering a therapeutic gene to the mouse brain that can result in long-term and widespread protein production. However, the human infant brain is more than 1000 times larger than the mouse brain, which will make the treatment of global neurometabolic disorders in children more difficult. In this study, we evaluated the ability of three AAV serotypes (1,2, and 5) to transduce cells in the cat brain as a model of a large mammalian brain. The human lysosomal enzyme beta-glucuronidase (GUSB) was used as a reporter gene, because it can be distinguished from feline GUSB by heat stability. The vectors were injected into the cerebral cortex, caudate nucleus, thalamus, corona radiata, internal capsule, and centrum semiovale of 8-week-old cats. The brains were evaluated for gene expression using in situ hybridization and enzyme histochemistry 10 weeks after surgery. The AAV2 vector was capable of transducing cells in the gray matter, while the AAV1 vector resulted in greater transduction of the gray matter than AAV2 as well as transduction of the white matter. AAV5 did not result in detectable transduction in the cat brain.

Indexed as

Models, AnimalAnimalsBrainCatsDependovirusGene ExpressionGenetic VectorsGlucuronidaseHistocytochemistryInjectionsIn Situ HybridizationSerotypingTransduction, GeneticGlucuronidase

Identifiers

PMID14502216
OpenAlexW2045403748

What OpenQuestion holds

Textmetadata
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.