Evidence map›Paper›PMID 36454475›Full record

ArticleAdvances in experimental medicine and biology2023

Enabling Precision Medicine with CRISPR-Cas Genome Editing Technology: A Translational Perspective.

Nazma F Ilahibaks, Maike J Hulsbos, Zhiyong Lei, Pieter Vader, Joost P G Sluijter

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In one paragraph

Article in Advances in experimental medicine and biology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
2.5field-weighted citation impact, top 10% 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

3 citing papers in PubMed, 5 citations in OpenAlex.

  1. Review
  2. Review
  3. Review
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 1 institution in 1 country.

Nazma F IlahibaksLaboratory of Experimental Cardiology, Department of Cardiology, University Medical Center, Utrecht, The Netherlands.
Maike J HulsbosLaboratory of Experimental Cardiology, Department of Cardiology, University Medical Center, Utrecht, The Netherlands.
Zhiyong LeiLaboratory of Experimental Cardiology, Department of Cardiology, University Medical Center, Utrecht, The Netherlands.
Pieter VaderLaboratory of Experimental Cardiology, Department of Cardiology, University Medical Center, Utrecht, The Netherlands.
Joost P G SluijterLaboratory of Experimental Cardiology, Department of Cardiology, University Medical Center, Utrecht, The Netherlands. J.Sluijter@umcutrecht.nl.
University Medical Center Utrecht · NL

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Genome editing technologies, particularly CRISPR-Cas (clustered regularly interspaced short palindromic repeats (CRISPR) associated nucleases), are redefining the boundaries of therapeutic gene therapy. CRISPR-Cas is a robust, straightforward, and programmable genome editing tool capable of mediating site-specific DNA modifications. The rapid advancements from discovery to clinical adaptation have expanded the therapeutic landscape to treat genetically defined diseases. Together with the technical developments in human DNA and RNA sequencing, CRISPR-directed gene therapy enables a new era to realize precision medicine where pathogenic mutations underlying monogenic disorders can potentially be corrected. Also, protective or therapeutic genomic alterations can be introduced as preventative or curative therapy. Despite its high therapeutic potential, CRISPR-Cas´ clinical translation is still in its infancy and is highly dependent on its efficiency, specificity in gene corrections, and cell-specific delivery. Therefore, this chapter focuses on the challenges and opportunities the CRISPR-Cas toolbox offers together with delivery vehicles to realize its use for therapeutic gene editing. Furthermore, we discuss the obstacles the CRISPR-Cas system faces for successful clinical translation and summarize its current clinical progress.

Indexed as

Gene EditingPrecision MedicineCRISPR-Cas SystemsEndonucleasesGenetic TherapyHumansEndonucleasesCRISPRGene therapyGenome editingPrecision medicineTranslational medicine

Identifiers

PMID36454475
OpenAlexW4310701758

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.