Evidence map›Paper›PMID 31972154›Full record

ReviewCell systems2020

CRISPR-Based Synthetic Transcription Factors In Vivo: The Future of Therapeutic Cellular Programming.

Matthew Pandelakis, Elizabeth Delgado, Mo R Ebrahimkhani

Abstract readReview
In one paragraph

Review in Cell systems, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 41 papers.

0numbers the graph read from it
0cells of the map it votes in
41citing 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

41 citing papers in PubMed.

  1. Review
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  5. Review
  6. CRISPRa-Mediated Increase of OPA1 Expression in Dominant Optic Atrophy.International journal of molecular sciences · 2025
    Article
  7. Article
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  19. Article
  20. CRISPR activation and interference as investigative tools in the cardiovascular system.The international journal of biochemistry & cell biology · 2023
    Article
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.

Matthew PandelakisSchool of Biological and Health Systems Engineering, Ira A. Fulton Schools of Engineering, Arizona State University, Tempe, AZ, USA.
Elizabeth DelgadoSchool of Biological and Health Systems Engineering, Ira A. Fulton Schools of Engineering, Arizona State University, Tempe, AZ, USA.
Mo R EbrahimkhaniSchool of Biological and Health Systems Engineering, Ira A. Fulton Schools of Engineering, Arizona State University, Tempe, AZ, USA; Department of Pathology, Division of Experimental Pathology, University of Pittsburgh, Pittsburgh, PA, USA; Pittsburgh Liver Research Center, University of Pittsburgh, Pittsburgh, PA, USA. Electronic address: mo.ebr@pitt.edu.

Funding

Recreating Human Developmental Hematopoietic Niches for Ex Vivo Biomanufacturing of Blood Stem CellsR01HL141805 · NHLBI · UNIVERSITY OF PITTSBURGH AT PITTSBURGH · PI Mo Reza Ebrahimkhani · 2019 to 2026
$2.6M
Integration of systems and synthetic biology to advance development of human tissues ex vivoR01EB028532 · NIBIB · UNIVERSITY OF PITTSBURGH AT PITTSBURGH · PI EBRAHIMKHANI, MO REZA · 2019 to 2022
$1.7M
CRISPR logic circuits for safer and controllable gene therapiesR01EB024562 · NIBIB · UNIVERSITY OF PITTSBURGH AT PITTSBURGH · PI KIANI, SAMIRA · 2018 to 2021
$1.7M
NHLBI NIH HHS R01 HL141805NIBIB NIH HHS R01 EB024562NIBIB NIH HHS R01 EB028532
6 · The paper itself

Abstract

Pinpoint control over endogenous gene expression in vivo has long been a fevered dream for clinicians and researchers alike. With the recent repurposing of programmable, RNA-guided DNA endonucleases from the CRISPR bacterial immune system, this dream is becoming a powerful reality. Engineered CRISPR/Cas9-based transcriptional regulators and epigenome editors have enabled researchers to perturb endogenous gene expression in vivo, allowing for the therapeutic reprogramming of cell and tissue behavior. For this technology to be of maximal use, a variety of technological hurdles still need to be addressed. Better understanding of the design principle controlling gene expression together with technologies that enable spatiotemporal control of transcriptional engineering are fundamental for rational design, improved efficacy, and ultimately safe translation to humans. In this review, we will discuss recent advances and integrative strategies that can help pave the path toward a new class of transcriptional therapeutics.

Indexed as

CRISPR-Cas SystemsGenetic EngineeringHumansTranscription FactorsTranscription FactorsCas9CRISPRepigenome editingin vivo cellular programingsynthetic gene circuitssynthetic transcription factor

Identifiers

PMID31972154
PMCPMC7175797

What OpenQuestion holds

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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.