Evidence map›Paper›PMID 33558455›Full record

ReviewSignal transduction and targeted therapy2021

Viral vector platforms within the gene therapy landscape.

Jote T Bulcha, Yi Wang, Hong Ma, Phillip W L Tai, Guangping Gao

Open access · goldAbstract readReview
In one paragraph

Review in Signal transduction and targeted therapy, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 745 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
745citing papers in PubMed, 2 pooled it
149.9field-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

745 citing papers in PubMed, 2 syntheses or guidelines pooled it, 1,255 citations in OpenAlex.

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  7. Retinoic acid in health and disease.Signal transduction and targeted therapy · 2026
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685 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

5 authors at 2 institutions in 2 countries.

Jote T Bulcha *Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA.
Yi Wang *Department of Pathophysiology, West China College of Basic medical sciences & Forensic Medicine, Sichuan University, Chengdu, China.
Hong MaHorae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA.
Phillip W L TaiHorae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA. phillip.tai2@umassmed.edu.ORCID http://orcid.org/0000-0001-7409-8344
Guangping GaoHorae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA. Guangping.Gao@umassmed.edu.
University of Massachusetts Chan Medical School · USWest China Medical Center of Sichuan University · CN

Funding

Project 4: A permanent off-switch for AAVU19AI149646 · NIAID · UNIVERSITY OF FLORIDA · PI FARZAN, MICHAEL R. · 2020 to 2024
$14.0M
Viral escape from AAV expressed transgenesP01AI100263 · NIAID · SCRIPPS FLORIDA · PI GAO, GUANGPING · 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 AI100263NIAID NIH HHS U19 AI149646NINDS NIH HHS R01 NS076991
6 · The paper itself

Abstract

Throughout its 40-year history, the field of gene therapy has been marked by many transitions. It has seen great strides in combating human disease, has given hope to patients and families with limited treatment options, but has also been subject to many setbacks. Treatment of patients with this class of investigational drugs has resulted in severe adverse effects and, even in rare cases, death. At the heart of this dichotomous field are the viral-based vectors, the delivery vehicles that have allowed researchers and clinicians to develop powerful drug platforms, and have radically changed the face of medicine. Within the past 5 years, the gene therapy field has seen a wave of drugs based on viral vectors that have gained regulatory approval that come in a variety of designs and purposes. These modalities range from vector-based cancer therapies, to treating monogenic diseases with life-altering outcomes. At present, the three key vector strategies are based on adenoviruses, adeno-associated viruses, and lentiviruses. They have led the way in preclinical and clinical successes in the past two decades. However, despite these successes, many challenges still limit these approaches from attaining their full potential. To review the viral vector-based gene therapy landscape, we focus on these three highly regarded vector platforms and describe mechanisms of action and their roles in treating human disease.

Indexed as

DependovirusGenetic TherapyGenetic VectorsGene Transfer TechniquesHumansLentivirus

Identifiers

PMID33558455
PMCPMC7868676
OpenAlexW3128825549

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.