Evidence map›Paper›PMID 20336156›Full record

ArticleGene therapy2010

Near-perfect infectivity of wild-type AAV as benchmark for infectivity of recombinant AAV vectors.

N Zeltner, E Kohlbrenner, N Clément, T Weber, R M Linden

Abstract read
In one paragraph

Article in Gene therapy, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 46 papers.

0numbers the graph read from it
0cells of the map it votes in
46citing papers in PubMed
–field-weighted citation impact
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

46 citing papers in PubMed.

  1. Distinct YY dinucleotide periodicity in adeno-associated virus DNA.Molecular therapy : the journal of the American Society of Gene Therapy · 2026
    Article
  2. Viral gene replication enhances AAV vector quality and reduces manufacturing costs.Molecular therapy : the journal of the American Society of Gene Therapy · 2026
    Article
  3. Article
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  5. Article
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  9. A clinician's guide to AAV production - How manufacturing platforms shape vector properties.Medizinische Genetik : Mitteilungsblatt des Berufsverbandes Medizinische Genetik e.V · 2025
    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

5 authors.

N ZeltnerDepartment of Gene and Cell Medicine, Mount Sinai School of Medicine, New York, NY, USA.
E Kohlbrenner
N Clément
T Weber
R M Linden

Funding

Viral Vector, Cell Line, Tissue & Mouse Model Production, Validation & ProcessingP01DK062345 · NIDDK · MOUNT SINAI SCHOOL OF MEDICINE OF NYU · PI WILSON, PATRICIA D. · 2003 to 2007
$6.7M
GROWTH, DIFFERENTIATION AND GENETIC ALTERATION OF HUMAN ES CELLSP20GM075019 · NIGMS · MOUNT SINAI SCHOOL OF MEDICINE OF NYU · PI KELLER, GORDON · 2005 to 2007
$2.8M
Altering AAV TropismR01HL077322 · NHLBI · MOUNT SINAI SCHOOL OF MEDICINE OF NYU · PI WEBER, THOMAS · 2005 to 2008
$1.5M
AAV-mediated targeted DNA integration in mouse ES cellsR01GM071023 · NIGMS · MOUNT SINAI SCHOOL OF MEDICINE OF NYU · PI LINDEN, RALPH MICHAEL · 2005 to 2008
$1.2M
NHLBI NIH HHS HL077322NHLBI NIH HHS R01 HL077322NIDDK NIH HHS DK062345NIDDK NIH HHS P01 DK062345NIGMS NIH HHS GM071023NIGMS NIH HHS GM075019NIGMS NIH HHS P20 GM075019NIGMS NIH HHS R01 GM071023
6 · The paper itself

Abstract

Viral vectors derived from adeno-associated viruses (AAVs) are widely used for gene transfer both in vitro and in vivo. The increasing use of AAV as a gene transfer vector, as well as recently shown immunological complications in clinical trials, highlight the necessity to define the specific activity of vector preparations beyond current standards. In this report, we determined the infectious, physical and genome-containing particle titers of several wild-type AAV type 2 (wtAAV2) and recombinant AAV type 2 (rAAV2) preparations that were produced and purified by standard methods. We found that the infectivity of wtAAV2 approaches a physical-to-infectious particle ratio of one. This near-perfect physical-to-infectious particle ratio defines a 'ceiling' for the theoretically achievable quality of recombinant AAV vectors. In comparison, for rAAV2, only approximately 50 out of 100 viral particles contained a genome and, more strikingly, only approximately 1 of the 100 viral particles was infectious. Our findings suggest that current strategies for rAAV vector design, production and/or purification should be amenable to improvements. Ultimately, this could result in the generation of near-perfect vector particles, a prospect with significant implications for gene therapy.

Indexed as

Genetic VectorsVirionDependovirusGenetic EngineeringGenetic TherapyGenome, ViralRecombination, Genetic

Identifiers

PMID20336156
PMCPMC2900506

What OpenQuestion holds

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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.