Evidence map›Paper›PMID 40114706›Full record

ArticleMolecular therapy. Nucleic acids2025

Non-invasive detection of allele-specific CRISPR-SaCas9-KKH disruption of

Katia E Maalouf, Dawn Madison Frederick, Nutan Sharma, Edwina Abou Haidar, Tianhe Xiao, Justin Seungkyu Han, Mohammed S Mahamdeh, Roy J Soberman, David Rufino-Ramos, Benjamin P Kleinstiver and 4 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Nucleic acids, 2025. 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. 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

14 authors.

Katia E MaaloufMolecular Neurogenetics Unit, Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Charlestown, MA 02129, USA.
Dawn Madison FrederickMolecular Neurogenetics Unit, Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Charlestown, MA 02129, USA.
Nutan SharmaThe Collaborative Center for X-linked Dystonia-Parkinsonism, Massachusetts General Hospital, Charlestown, MA 02129, USA.
Edwina Abou HaidarMolecular Neurogenetics Unit, Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Charlestown, MA 02129, USA.
Tianhe XiaoMolecular Neurogenetics Unit, Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Charlestown, MA 02129, USA.
Justin Seungkyu HanThe Collaborative Center for X-linked Dystonia-Parkinsonism, Massachusetts General Hospital, Charlestown, MA 02129, USA.
Mohammed S MahamdehDivision of Cardiology, Harvard Medical School, Boston, MA, USA.
Roy J SobermanMass General Brigham Center of Excellence for Molecular Imaging, Charlestown, MA 02129, USA.
David Rufino-RamosCenter for Genomic Medicine, Massachusetts General Hospital, Boston, MA 02114, USA.
Benjamin P KleinstiverCenter for Genomic Medicine, Massachusetts General Hospital, Boston, MA 02114, USA.
Hyder A JinnahDepartments of Neurology and Human Genetics, Emory University School of Medicine, Atlanta, GA 30322, USA.
Christine A VaineThe Collaborative Center for X-linked Dystonia-Parkinsonism, Massachusetts General Hospital, Charlestown, MA 02129, USA.
D Cristopher BraggThe Collaborative Center for X-linked Dystonia-Parkinsonism, Massachusetts General Hospital, Charlestown, MA 02129, USA.
Koen BreyneMolecular Neurogenetics Unit, Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Charlestown, MA 02129, USA.

Funding

Scalable Development of Custom Genome Editing TechnologiesDP2CA281401 · NCI · MASSACHUSETTS GENERAL HOSPITAL · PI KLEINSTIVER, BENJAMIN PETER · 2022 to 2025
$2.5M
Boosting IL-12-induced anti-glioblastoma activity via immunotherapeutic extracellular vesicles.K22CA282019 · NCI · MASSACHUSETTS GENERAL HOSPITAL · PI Koen Breyne · 2024 to 2026
$482k
NCI NIH HHS DP2 CA281401NCI NIH HHS K22 CA282019
6 · The paper itself

Abstract

DYT1 dystonia is a neurological movement disorder characterized by a dominant 3-base pair deletion (ΔGAG) in the

Indexed as

adeno-associated virus vectorCRISPR/Cas gene editingdGAGDYT1 dystoniaexRNAextracellular vesiclesMT: RNA/DNA EditingSaCas9-KKHtheranosticTOR1A

Identifiers

PMID40114706
PMCPMC11925580

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.