Evidence map›Paper›PMID 30783968›Full record

ArticleMethods in molecular biology (Clifton, N.J.)2019

Quantitative and Digital Droplet-Based AAV Genome Titration.

Julio Sanmiguel, Guangping Gao, Luk H Vandenberghe

Open access · greenAbstract read
In one paragraph

Article in Methods in molecular biology (Clifton, N.J.), 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 47 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
47citing papers in PubMed, 1 pooled it
13.3field-weighted citation impact, top 1% 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

47 citing papers in PubMed, 1 synthesis or guideline pooled it, 68 citations in OpenAlex.

  1. What's new and what's next for gene therapy in Pompe disease?Expert opinion on biological therapy · 2022
    Pooled it
  2. Article
  3. Lung endothelial cytopathic tau is sufficient to impair long-term potentiation during infection.American journal of respiratory cell and molecular biology · 2026
    Article
  4. Article
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  6. bioRxiv : the preprint server for biology · 2026
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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

3 authors at 2 institutions in 1 country.

Julio SanmiguelGrousbeck Gene Therapy Center, Schepens Eye Research Institute and Massachusetts Eye and Ear Infirmary, Boston, MA, USA.
Guangping GaoHorae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA. Guangping.Gao@umassmed.edu.
Luk H VandenbergheGrousbeck Gene Therapy Center, Schepens Eye Research Institute and Massachusetts Eye and Ear Infirmary, Boston, MA, USA. Luk_Vandenberghe@meei.harvard.edu.
Harvard University · USUniversity of Massachusetts Chan Medical School · US

Funding

Viral escape from AAV expressed transgenesP01AI100263 · NIAID · SCRIPPS FLORIDA · PI FARZAN, MICHAEL R. · 2012 to 2016
$11.9M
Viral Vector CoreP01HL131471 · NHLBI · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI FLOTTE, TERENCE R. · 2016 to 2020
$11.3M
Oligodendrocyte-focused rAAV gene therapy strategies for Canavan disease and LeukodystrophiesR01NS076991 · NINDS · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI Guangping Gao · 2012 to 2026
$5.7M
Next Generation of Recombinant AAV Serotype Vectors for Gene TherapyR01HL097088 · NHLBI · UNIVERSITY OF FLORIDA · PI GAO, GUANGPING, HERZOG, ROLAND W. · 2010 to 2018
$5.4M
Develop combinatorial non-viral and viral CRISPR delivery for lung diseasesUG3HL147367 · NHLBI · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI ANDERSON, DANIEL G, GAO, GUANGPING · 2018 to 2020
$2.7M
NHLBI NIH HHS P01 HL131471NHLBI NIH HHS R01 HL097088NHLBI NIH HHS UG3 HL147367NIAID NIH HHS P01 AI100263NINDS NIH HHS R01 NS076991
6 · The paper itself

Abstract

The adeno-associated viral vector (AAV) platform has developed into a primary modality for efficient in vivo, and in more limited settings, in vitro or ex vivo gene transfer. Its applications range from a tool for experimental purposes to preclinical and clinical gene therapy. The ability to accurately and reproducibly quantify vector concentration is critical for any of these applications. While several quantification assays are available, here we outline a detailed protocol for the quantification of DNase-I protected vector genomes reliant on the polymerase chain reaction (PCR) as a measure of the active component of the vector, namely its transgene cargo. With the emergence of droplet digital PCR (ddPCR), we provide side-by-side protocols for traditional TaqMan™ real-time, quantitative PCR (qPCR) and ddPCR, as well as comparative data generated with both methods. Lastly, we discuss the importance of the use of surfactant (here, Pluronic

Indexed as

Genome, ViralReal-Time Polymerase Chain ReactionViral LoadDependovirusGenetic VectorsHumansSensitivity and SpecificityWorkflowAAVAdeno-associated virusddPCRDroplet digital PCRGenomeqPCRQuantitative PCRReal-time PCRTitrationVector

Identifiers

PMID30783968
PMCPMC7377043
OpenAlexW2916895573

What OpenQuestion holds

Textmetadata
LicenceTDM
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.