Evidence map›Paper›PMID 39781718›Full record

ReviewCurrent gene therapy2025

CRISPR/Cas System: A Powerful Strategy to Improve Monogenic Human Diseases as Therapeutic Delivery; Current Applications and Challenges.

Fatemeh Saberi, Zeinab Yousefi-Najafabadi, Forough Shams, Zeinab Dehghan, Sepideh Ahmadi, Tayyebeh Pilehchi, Effat Noori, Zeinab Esmaeelzadeh, Maryam Bazgiri, Rezvan Mohammadi and 7 more

Abstract readReview
PubMed Publisher
In one paragraph

Review in Current gene therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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

2 citing papers in PubMed.

  1. Review
  2. 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

17 authors.

Fatemeh SaberiDepartment of Medical Biotechnology, School of Advanced Technologies in Medicine, Shahid Beheshti University of Medical Sciences, 1968917313, Tehran, Iran.
Zeinab Yousefi-NajafabadiDepartment of Medical Biotechnology, School of Advanced Technologies in Medicine, Tehran University of Medical Sciences, 5469775141, Tehran, Iran.
Forough ShamsDepartment of Medical Biotechnology, School of Advanced Technologies in Medicine, Shahid Beheshti University of Medical Sciences, 1968917313, Tehran, Iran.
Zeinab DehghanDepartment of Comparative Biomedical Sciences, School of Advanced Medical Sciences and Technologies, Shiraz University of Medical Sciences, 7134874478, Shiraz, Iran.
Sepideh AhmadiDepartment of Medical Biotechnology, School of Advanced Technologies in Medicine, Shahid Beheshti University of Medical Sciences, 1968917313, Tehran, Iran.
Tayyebeh PilehchiDepartment of Medical Biotechnology, School of Advanced Technologies in Medicine, Shahid Beheshti University of Medical Sciences, 1968917313, Tehran, Iran.
Effat NooriDepartment of Biotechnology, Faculty of Medicine, Shahed University, 3319118651, Tehran, Iran.
Zeinab EsmaeelzadehDepartment of Medical Biotechnology, School of Advanced Technologies in Medicine, Shahid Beheshti University of Medical Sciences, 1968917313, Tehran, Iran.
Maryam BazgiriDepartment of Animal Science, Agriculture and Natural Resources University of Khuzestan, 6341773637, Ahvaz, Iran.
Rezvan MohammadiDepartment of Medical Biotechnology, School of Advanced Technologies in Medicine, Shahid Beheshti University of Medical Sciences, 1968917313, Tehran, Iran.
Farzaneh KhaniDepartment of Stem Cell and Regenerative Medicine, Institute of Medical Biotechnology, National Institute of Genetic Engineering & Biotechnology (NIGEB), 14965161, Tehran, Iran.
Marzieh SameniDepartment of Medical Biotechnology, School of Advanced Technologies in Medicine, Shahid Beheshti University of Medical Sciences, 1968917313, Tehran, Iran.
Parisa MoradbeigiDepartment of Clinical Science, School of Veterinary Medicine, Shiraz University, 7144169155, Shiraz, Iran.
Gholam Ali KardarDepartment of Medical Biotechnology, School of Advanced Technologies in Medicine, Tehran University of Medical Sciences, 5469775141, Tehran, Iran.
Mohammad SalehiDepartment of Medical Biotechnology, School of Advanced Technologies in Medicine, Shahid Beheshti University of Medical Sciences, 1968917313, Tehran, Iran.
Yong TengDepartment of Hematology and Medical Oncology, Winship Cancer Institute, Emory University, Atlanta, GA 30322, USA.
Vahid JajarmiDepartment of Medical Biotechnology, School of Advanced Technologies in Medicine, Shahid Beheshti University of Medical Sciences, 1968917313, Tehran, Iran.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The 5,000 to 8,000 monogenic diseases are inherited disorders leading to mutations in a single gene. These diseases usually appear in childhood and sometimes lead to morbidity or premature death. Although treatments for such diseases exist, gene therapy is considered an effective and targeted method and has been used in clinics for monogenic diseases since 1989. Monogenic diseases are good candidates for novel therapeutic technologies like gene editing approaches to repair gene mutations. Clustered regularly interspaced short palindromic repeats (CRISPR)-based systems, the pioneer and effective gene editing tool, are utilized for

Indexed as

CRISPR-Cas SystemsGene EditingGenetic Diseases, InbornGenetic TherapyAnimalsHumansCRISPR/Cas systemex vivo modelsgene editinggene therapyin vivo modelsmonogenic diseases.

Identifiers

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.