Evidence map›Paper›PMID 31968213›Full record

ReviewMolecular therapy : the journal of the American Society of Gene Therapy2020

Immune Responses to Viral Gene Therapy Vectors.

Jamie L Shirley, Ype P de Jong, Cox Terhorst, Roland W Herzog

Open access · bronzeAbstract readReview
In one paragraph

Review in Molecular therapy : the journal of the American Society of Gene Therapy, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 452 papers, 3 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
452citing papers in PubMed, 3 pooled it
69.1field-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

452 citing papers in PubMed, 3 syntheses or guidelines pooled it, 724 citations in OpenAlex.

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392 more citing papers are in PubMed but not listed here.

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 4 institutions in 1 country.

Jamie L ShirleyGene Therapy Center, University of Massachusetts, Worchester, MA, USA.
Ype P de JongDivision of Gastroenterology and Hepatology, Weill Cornell Medicine, New York, NY, USA.
Cox TerhorstDivision of Immunology, Beth Israel Deaconess Medical Center (BIDMC), Harvard Medical School, Boston, MA, USA.
Roland W HerzogHerman B Wells Center for Pediatric Research, Indiana University School of Medicine, Indianapolis, IN, USA. Electronic address: rwherzog@iu.edu.
Beth Israel Deaconess Medical Center · USCornell University · USIndiana University School of MedicineUniversity of Massachusetts Chan Medical School · US

Funding

Enhancing immune regulation in gene therapy for hemophiliaR01HL131093 · NHLBI · UNIVERSITY OF FLORIDA · PI Ype Peter De Jong, Roland W. Herzog · 2016 to 2026
$7.2M
Immunology of Factor IX Gene Transfer to LiverR01AI051390 · NIAID · UNIVERSITY OF FLORIDA · PI HERZOG, ROLAND W. · 2002 to 2022
$6.7M
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
Modeling alcohol toxicity in human hepatocytesR01AA027327 · NIAAA · WEILL MEDICAL COLL OF CORNELL UNIV · PI DE JONG, YPE PETER · 2019 to 2023
$1.9M
NHLBI NIH HHS R01 HL097088NHLBI NIH HHS R01 HL131093NIAAA NIH HHS R01 AA027327NIAID NIH HHS R01 AI051390
6 · The paper itself

Abstract

Several viral vector-based gene therapy drugs have now received marketing approval. A much larger number of additional viral vectors are in various stages of clinical trials for the treatment of genetic and acquired diseases, with many more in pre-clinical testing. Efficiency of gene transfer and ability to provide long-term therapy make these vector systems very attractive. In fact, viral vector gene therapy has been able to treat or even cure diseases for which there had been no or only suboptimal treatments. However, innate and adaptive immune responses to these vectors and their transgene products constitute substantial hurdles to clinical development and wider use in patients. This review provides an overview of the type of immune responses that have been documented in animal models and in humans who received gene transfer with one of three widely tested vector systems, namely adenoviral, lentiviral, or adeno-associated viral vectors. Particular emphasis is given to mechanisms leading to immune responses, efforts to reduce vector immunogenicity, and potential solutions to the problems. At the same time, we point out gaps in our knowledge that should to be filled and problems that need to be addressed going forward.

Indexed as

ImmunityAdaptive ImmunityAnimalsAntigen-Presenting CellsGenetic VectorsHost-Pathogen InteractionsHumansImmune ToleranceImmunity, InnateSignal TransductionVirusesadaptive immunityadeno-associated virusadenovirusinnate immunitylentivirus

Identifiers

PMID31968213
PMCPMC7054714
OpenAlexW2999858469

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.