Evidence map›Paper›PMID 12552020›Full record

ArticleJournal of virology2003

Characterization of tissue tropism determinants of adeno-associated virus type 1.

Bernd Hauck, Weidong Xiao

Open access · greenAbstract read
In one paragraph

Article in Journal of virology, 2003. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 58 papers.

0numbers the graph read from it
0cells of the map it votes in
58citing papers in PubMed
5.3field-weighted citation impact, top 4% 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

58 citing papers in PubMed, 120 citations in OpenAlex.

  1. Tissue-specific gene delivery approaches.Bioengineering & translational medicine · 2026
    Review
  2. Review
  3. AAV vector development, back to the future.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Review
  4. RNA Structure: Past, Future, and Gene Therapy Applications.International journal of molecular sciences · 2024
    Review
  5. Identification of a novel neutralization epitope in rhesus AAVs.Molecular therapy. Methods & clinical development · 2024
    Article
  6. Bone Growth Induction in Mucopolysaccharidosis IVA Mouse.International journal of molecular sciences · 2023
    Article
  7. Review
  8. AAV vectors: The Rubik's cube of human gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2022
    Review
  9. Review
  10. Review
  11. Review
  12. Review
  13. Article
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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

2 authors at 2 institutions in 1 country.

Bernd HauckDepartment of Pediatrics, University of Pennsylvania Medical Center, Philadelphia, PA 19104, USA.
Weidong Xiao
Children's Hospital of Philadelphia · USUniversity of Pennsylvania · US

Funding

Molecular and Cellular Characterization of AAV1 VectorR01HL069051 · NHLBI · CHILDRENS HOSPITAL OF PHILADELPHIA · PI XIAO, WEIDONG · 2002 to 2005
$1.3M
NHLBI NIH HHS R01 HL 069051NHLBI NIH HHS R01 HL069051
6 · The paper itself

Abstract

Muscle is an attractive target for gene delivery because of its mass and because vectors can be delivered in a noninvasive fashion. Adeno-associated virus (AAV) has been shown to be effective for muscle-targeted gene transfer. Recent progress in characterization of AAV serotype 1 (AAV1) and AAV6 demonstrated that these two AAV serotypes are far more efficient in transducing muscle than is the traditionally used AAV2. Since all cis elements are identical in these vectors, the potential determinants for their differences in transducing muscle appear to be located within the AAV capsid proteins. In the present study, a series of AAV capsid mutants were generated to identify the major regions affecting AAV transduction efficiency in muscle. Replacement of amino acids 350 to 736 of AAV2 VP1 with the corresponding amino acids from VP1 of AAV1 resulted in a hybrid vector that behaved very similarly to AAV1 in vitro and in vivo in muscle. Characterization of additional mutants carrying smaller regions of the AAV1 VP1 amino acid sequence in the AAV2 capsid protein suggested that amino acids 350 to 430 of VP1 function as a major tissue tropism determinant. Further analysis showed that the heparin binding domain and the major antigenic determinants in the AAV capsid region were not necessary for the efficiency of AAV1 transduction of muscle.

Indexed as

Transduction, GeneticAmino Acid SequenceCapsid ProteinsDependovirusGenetic VectorsHumansMolecular Sequence DataMusclesMutationCapsid Proteins

Identifiers

PMID12552020
PMCPMC141099
OpenAlexW1986428879

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.