Evidence map›Paper›PMID 41496894›Full record

ReviewMolecular therapy. Nucleic acids2026

CRISPR-Cas editing technologies for viral-mediated gene therapies of human diseases: Mechanisms, progress, and challenges.

Boris Kantor, Leanne Duke, Pradeep G Bhide

Abstract readReview
In one paragraph

Review in Molecular therapy. Nucleic acids, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. Genome editing between wonder and rejection.Frontiers in bioengineering and biotechnology · 2026
    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.

Boris KantorViral Vector and Gene Editing Cores, Florida Institute for Pediatric Rare Diseases, College of Medicine, Florida State University, Tallahassee, FL 32306, USA.
Leanne DukeViral Vector and Gene Editing Cores, Florida Institute for Pediatric Rare Diseases, College of Medicine, Florida State University, Tallahassee, FL 32306, USA.
Pradeep G BhideFlorida Institute for Pediatric Rare Diseases, College of Medicine, Florida State University, Tallahassee, FL 32306, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The gene therapy landscape has evolved substantially in recent years, beginning with the approval of the first adeno-associated virus-based gene therapy, Luxterna, in 2017. Since then, the US FDA has approved nearly 30 new viral gene therapy programs, with notable examples including Zolgensma, Spinraza, Hemgenix, Zynteglo, Lyfgenia, Kymriah, Skysona, and Tecelra. Remarkably, all these products rely on delivery via adeno-associated vectors (AAVs) and lentiviral vectors (LVs). Improvements in viral-mediated gene transfer efficiency and clinical-scale manufacturing, together with immense commercial interest, have greatly propelled the clinical adoption of gene therapy products. In recent years, clustered regularly interspaced short palindromic repeats (CRISPR) and its related Cas proteins (CRISPR-Cas) have made significant advances in gene therapy, offering next-generation approaches for curative gene editing to treat genetic diseases and disorders. In this review, we examine the range of these therapeutics and their viral carriers, focusing primarily on LVs and AAVs. We provide a snapshot of the current status of the field and highlight some of the current challenges in the clinical application of gene therapy, with particular emphasis on viral CRISPR-Cas-based technologies and their future potential.

Indexed as

AAVadeno-associated vectorsall-in-one delivery systembase editingclinical trialsclustered regularly-interspaced short palindromic repeatsCRISPR-associated protein 9CRISPR-Cas9epigenome-based editinggene deliverygene therapylentiviral vectorsMT: Delivery Strategiesprime editing

Identifiers

PMID41496894
PMCPMC12767858

What OpenQuestion holds

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Registered trials

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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.