Evidence map›Paper›PMID 24917359›Full record

ArticleExpert opinion on therapeutic targets2014

Activating human genes with zinc finger proteins, transcription activator-like effectors and CRISPR/Cas9 for gene therapy and regenerative medicine.

Charles A Gersbach, Pablo Perez-Pinera

Open access · bronzeAbstract readEditorial
In one paragraph

Article in Expert opinion on therapeutic targets, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 29 papers.

0numbers the graph read from it
0cells of the map it votes in
29citing papers in PubMed
3.4field-weighted citation impact, top 7% 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

29 citing papers in PubMed, 49 citations in OpenAlex.

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  13. Boosting, Not Breaking: CRISPR Activators Treat Disease Models.Molecular therapy : the journal of the American Society of Gene Therapy · 2018
    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

2 authors at 2 institutions in 1 country.

Charles A GersbachDuke University, Department of Biomedical Engineering , Room 136 Hudson Hall, Box 90281, Durham, NC 27708-0281 , USA +1 919 613 2147 ; +1 919 668 0795 ; charles.gersbach@duke.edu.
Pablo Perez-Pinera
Duke Medical Center · USDuke University · US

Funding

Engineering Targeted Epigenetic Modifiers for Precise Control of Gene RegulationR01DA036865 · NIDA · DUKE UNIVERSITY · PI CRAWFORD, GREGORY E, GERSBACH, CHARLES A. · 2013 to 2017
$2.6M
Engineering Morphogenetic Factors for Enhanced Genetic ReprogrammingDP2OD008586 · OD · DUKE UNIVERSITY · PI GERSBACH, CHARLES A. · 2011 to 2011
$2.4M
Spatially Controlled Gene Delivery of Morphogenetic Factors from Woven ScaffoldsR03AR061042 · NIAMS · DUKE UNIVERSITY · PI GERSBACH, CHARLES A. · 2011 to 2013
$224k
NIAMS NIH HHS R03 AR061042NIAMS NIH HHS R03AR061042NIDA NIH HHS R01 DA036865NIDA NIH HHS R01DA036865NIH HHS DP2 OD008586NIH HHS DP2OD008586
6 · The paper itself

Abstract

New technologies have recently been developed to control the expression of human genes in their native genomic context by engineering synthetic transcription factors that can be targeted to any DNA sequence. The ability to precisely regulate any gene as it occurs naturally in the genome provides a means to address a variety of diseases and disorders. This approach also circumvents some of the traditional challenges of gene therapy. In this editorial, we review the technologies that have enabled targeted human gene activation, including the engineering of transcription factors based on zinc finger proteins, transcription activator-like effectors and the CRISPR/Cas9 system. Additionally, we highlight examples in which these methods have been developed for therapeutic applications and discuss challenges and opportunities.

Indexed as

Base SequenceClustered Regularly Interspaced Short Palindromic RepeatsGene Expression RegulationGenetic TherapyHumansRegenerative MedicineTrans-ActivatorsTranscription FactorsZinc FingersTrans-ActivatorsTranscription FactorsCas9CRISPRgene editinggene regulationgene therapygenetic reprogrammingprotein engineeringtranscription activator-like effectortranscription factorzinc finger

Identifiers

PMID24917359
PMCPMC4236187
OpenAlexW2029202844

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.