Evidence map›Paper›PMID 29130351›Full record

ArticleHuman gene therapy. Clinical development2017

The Gene Therapy Resource Program: A Decade of Dedication to Translational Research by the National Heart, Lung, and Blood Institute.

Terence R Flotte, Eric Daniels, Janet Benson, Jeneé M Bevett-Rose, Kenneth Cornetta, Margaret Diggins, Julie Johnston, Susan Sepelak, Johannes C M van der Loo, James M Wilson and 1 more

Abstract read
In one paragraph

Article in Human gene therapy. Clinical development, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing 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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

11 authors.

Terence R Flotte1 Horae Gene Therapy Center, University of Massachusetts Medical School , Worcester, Massachusetts.
Eric Daniels2 Social and Scientific Systems, Inc. , Silver Spring, Maryland.
Janet Benson3 Lovelace Biomedical and Environmental Research Institute , Albuquerque, New Mexico.
Jeneé M Bevett-Rose2 Social and Scientific Systems, Inc. , Silver Spring, Maryland.
Kenneth Cornetta4 Department of Medical and Molecular Genetics, Indiana University , Indianapolis, Indiana.
Margaret Diggins2 Social and Scientific Systems, Inc. , Silver Spring, Maryland.
Julie Johnston5 Gene Therapy Program, Department of Medicine, University of Pennsylvania , Philadelphia, Pennsylvania.
Susan Sepelak2 Social and Scientific Systems, Inc. , Silver Spring, Maryland.
Johannes C M van der Loo5 Gene Therapy Program, Department of Medicine, University of Pennsylvania , Philadelphia, Pennsylvania.
James M Wilson5 Gene Therapy Program, Department of Medicine, University of Pennsylvania , Philadelphia, Pennsylvania.
Cheryl L McDonald7 The National Heart, Lung and Blood Institute , Bethesda, Maryland.

Funding

Viral Vector CoreP01HL131471 · NHLBI · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI XUE, WEN · 2016 to 2020
$11.3M
NHLBI NIH HHS P01 HL131471
6 · The paper itself

Abstract

Over a 10-year period, the Gene Therapy Resource Program (GTRP) of the National Heart Lung and Blood Institute has provided a set of core services to investigators to facilitate the clinical translation of gene therapy. These services have included a preclinical (research-grade) vector production core; current Good Manufacturing Practice clinical-grade vector cores for recombinant adeno-associated virus and lentivirus vectors; a pharmacology and toxicology core; and a coordinating center to manage program logistics and to provide regulatory and financial support to early-phase clinical trials. In addition, the GTRP has utilized a Steering Committee and a Scientific Review Board to guide overall progress and effectiveness and to evaluate individual proposals. These resources have been deployed to assist 82 investigators with 172 approved service proposals. These efforts have assisted in clinical trial implementation across a wide range of genetic, cardiac, pulmonary, and blood diseases. Program outcomes and potential future directions of the program are discussed.

Indexed as

Anniversaries and Special EventsDependovirusGenetic TherapyGenetic VectorsHumansLentivirusNational Heart, Lung, and Blood Institute (U.S.)Translational Research, BiomedicalUnited Statesadeno-associated virusclinical trialgene therapylentiviruspreclinicalvector

Identifiers

PMID29130351
PMCPMC5733658

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.