Evidence map›Paper›PMID 39640016›Full record

ArticleMolecular therapy. Nucleic acids2024

Rescue of the endogenous FVIII expression in hemophilia A mice using CRISPR-Cas9 mRNA LNPs.

Chun-Yu Chen, Xiaohe Cai, Barbara A Konkle, Carol H Miao

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Nucleic acids, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing 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

4 citing papers in PubMed.

  1. Advancements in CRISPR-basedFrontiers in genome editing · 2026
    Review
  2. Review
  3. Review
  4. Recent Advances in Gene Therapy for Hemophilia.Clinical and applied thrombosis/hemostasis : official journal of the International Academy of Clinical and Applied Thrombosis/Hemostasis
    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

4 authors.

Chun-Yu ChenSeattle Children's Research Institute, Seattle, WA 98101, USA.
Xiaohe CaiSeattle Children's Research Institute, Seattle, WA 98101, USA.
Barbara A KonkleWashington Center for Bleeding Disorders, Seattle, WA 98101, USA.
Carol H MiaoSeattle Children's Research Institute, Seattle, WA 98101, USA.

Funding

Ultrasound-mediated gene delivery to achieve therapeutic correction of hemophilia AR01HL151077 · NHLBI · SEATTLE CHILDREN'S HOSPITAL · PI MIAO, CAROL H · 2020 to 2023
$3.2M
Gene Editing for Hemophilia A Treatment Using Lipid NanoparticlesR01HL169793 · NHLBI · SEATTLE CHILDREN'S HOSPITAL · PI Carol H Miao · 2024 to 2026
$2.2M
NHLBI NIH HHS R01 HL151077NHLBI NIH HHS R01 HL169793
6 · The paper itself

Abstract

Gene editing provides a promising alternative approach that may achieve long-term FVIII expression for hemophilia A (HemA) treatment. In this study, we investigated

Indexed as

CRISPR-Cas9factor VIIIgene editinghemophilia Aindel editinglipid nanoparticleliver sinusoidal endothelial cellsMT: RNA/DNA Editingnonviral gene therapy

Identifiers

PMID39640016
PMCPMC11617921

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.