Evidence map›Paper›PMID 36499601›Full record

ReviewInternational journal of molecular sciences2022

Induced Pluripotent Stem Cells and Genome-Editing Tools in Determining Gene Function and Therapy for Inherited Retinal Disorders.

Daniela Benati, Amy Leung, Pedro Perdigao, Vasileios Toulis, Jacqueline van der Spuy, Alessandra Recchia

Open access · goldAbstract readReview
In one paragraph

Review in International journal of molecular sciences, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.

0numbers the graph read from it
0cells of the map it votes in
5citing papers in PubMed
2.4field-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

5 citing papers in PubMed, 16 citations in OpenAlex.

  1. Review
  2. Article
  3. Correction of Griscelli Syndrome Type 2 causing mutations in theTurkish journal of biology = Turk biyoloji dergisi · 2024
    Article
  4. Review
  5. 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

6 authors at 3 institutions in 3 countries.

Daniela BenatiCentre for Regenerative Medicine, Department of Life Sciences, University of Modena and Reggio Emilia, 41125 Modena, Italy.ORCID 0000-0002-6599-7193
Amy LeungUCL Institute of Ophthalmology, London EC1V 9EL, UK.
Pedro PerdigaoCenter for Neuroscience and Cell Biology, University of Coimbra, 3004-504 Coimbra, Portugal.
Vasileios ToulisUCL Institute of Ophthalmology, London EC1V 9EL, UK.
Jacqueline van der SpuyUCL Institute of Ophthalmology, London EC1V 9EL, UK.
Alessandra RecchiaCentre for Regenerative Medicine, Department of Life Sciences, University of Modena and Reggio Emilia, 41125 Modena, Italy.
University College London · GBUniversity of Modena and Reggio Emilia · ITUniversity of Coimbra · PT

Funding

Fight for Sight 5129/5130Medical Research Council MR/P02582X/1
6 · The paper itself

Abstract

Inherited retinal disorders (IRDs) affect millions of people worldwide and are a major cause of irreversible blindness. Therapies based on drugs, gene augmentation or transplantation approaches have been widely investigated and proposed. Among gene therapies for retinal degenerative diseases, the fast-evolving genome-editing CRISPR/Cas technology has emerged as a new potential treatment. The CRISPR/Cas system has been developed as a powerful genome-editing tool in ophthalmic studies and has been applied not only to gain proof of principle for gene therapies in vivo, but has also been extensively used in basic research to model diseases-in-a-dish. Indeed, the CRISPR/Cas technology has been exploited to genetically modify human induced pluripotent stem cells (iPSCs) to model retinal disorders in vitro, to test in vitro drugs and therapies and to provide a cell source for autologous transplantation. In this review, we will focus on the technological advances in iPSC-based cellular reprogramming and gene editing technologies to create human in vitro models that accurately recapitulate IRD mechanisms towards the development of treatments for retinal degenerative diseases.

Indexed as

Induced Pluripotent Stem CellsRetinal DegenerationCRISPR-Cas SystemsGene EditingGenetic TherapyHumansCRISPR/Casinduced pluripotent stem cellsretinal disorders

Identifiers

PMID36499601
PMCPMC9735568
OpenAlexW4311629910

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.