Evidence map›Paper›PMID 21204705›Full record

ArticleHuman gene therapy2011

Evaluation of adeno-associated viral vectors for liver-directed gene transfer in dogs.

Peter Bell, Guangping Gao, Mark E Haskins, Lili Wang, Meg Sleeper, Huan Wang, Roberto Calcedo, Luk H Vandenberghe, Shu-Jen Chen, Chick Weisse and 2 more

Open access · greenAbstract readEvaluation Study
In one paragraph

Article in Human gene therapy, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 34 papers.

0numbers the graph read from it
0cells of the map it votes in
34citing papers in PubMed
3.0field-weighted citation impact, top 9% 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

34 citing papers in PubMed, 45 citations in OpenAlex.

  1. Trial
  2. Truncated complement factor H Y402 gene therapy rescues C3 glomerulonephritis.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Article
  3. AAV vector development, back to the future.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Review
  4. Article
  5. Article
  6. Article
  7. Review
  8. Exomap1 mouse: A transgenic model forExtracellular vesicle · 2023
    Article
  9. Article
  10. Article
  11. Article
  12. Article
  13. Article
  14. Review
  15. Review
  16. Article
  17. Article
  18. Review
  19. Article
  20. Article
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

12 authors at 1 institution in 1 country.

Peter BellGene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Guangping Gao
Mark E Haskins
Lili Wang
Meg Sleeper
Huan Wang
Roberto Calcedo
Luk H Vandenberghe
Shu-Jen Chen
Chick Weisse
Elanor Withnall
James M Wilson
University of Pennsylvania · US

Funding

Vector CoreP30DK047757 · NIDDK · UNIVERSITY OF PENNSYLVANIA · PI WILSON, JAMES M · 1993 to 2017
$18.0M
X-LINKED SEVERE COMBINED IMMUNODEFICIENCY IN THE DOGP40RR002512 · NCRR · UNIVERSITY OF PENNSYLVANIA · PI HASKINS, MARK E · 1985 to 2011
$7.6M
Gene Therapy of Mucopolysaccharidosis VIIR01DK054481 · NIDDK · UNIVERSITY OF PENNSYLVANIA · PI CASAL, MARGRET L · 1998 to 2017
$7.0M
NCRR NIH HHS P40 RR02512NIDDK NIH HHS DK54481NIDDK NIH HHS P30 DK47757
6 · The paper itself

Abstract

This study evaluated six adeno-associated viral (AAV) vectors expressing green fluorescent protein (GFP) from the liver-specific thyroid hormone-binding globulin (TBG) promoter made with novel capsids in canine liver-directed gene transfer. Studies in 1.5-month-old dogs, which were administered vector through a peripheral vein, showed that AAV8 capsid vectors had the most favorable performance profiles. Interestingly, the absolute levels of hepatocyte transduction achieved with AAV8 were lower in dogs compared with what had been achieved in mice and nonhuman primates. Additional studies were performed with AAV8 delivered into the hepatic artery in adult dogs, with higher doses of vector used to assess potential dose-limiting toxicities. These studies showed good transduction on day 7 in one dog that apparently was lost by day 28 in another dog through the generation of GFP-specific T cells. Each adult dog was carefully monitored for any hemodynamic changes associated with vector infusion. Both animals demonstrated mild to moderate hypotension and bradycardia, which appeared to be anesthesia-related, making it difficult to evaluate contributions of the vector.

Indexed as

AnimalsDependovirusDogsGenetic VectorsGene Transfer TechniquesGreen Fluorescent ProteinsHepatic ArteryLiverT-Lymphocytes, CytotoxicGreen Fluorescent Proteins

Identifiers

PMID21204705
PMCPMC3159528
OpenAlexW1978072154

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.