Evidence map›Paper›PMID 40649847›Full record

ArticleInternational journal of molecular sciences2025

Preclinical Evaluation of the Systemic Safety, Efficacy, and Biodistribution of a Recombinant AAV8 Vector Expressing FIX-TripleL in Hemophilia B Mice: Implications for Human Gene Therapy.

Sheng-Chieh Chou, Cheng-Po Huang, Ying-Hui Su, Chih-Hsiang Yu, Yung-Li Yang, Ssu-Chia Wang, Yi-Hsiu Lin, Yen-Ting Chen, Jia-Yi Li, Yen-Ting Chang and 2 more

Abstract read
In one paragraph

Article in International journal of molecular sciences, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

12 authors.

Sheng-Chieh ChouDepartment of Internal Medicine, National Taiwan University Hospital, College of Medicine, National Taiwan University, Taipei 100, Taiwan.ORCID 0000-0002-4121-019X
Cheng-Po HuangTrineo Biotechnology Co., Ltd., New Taipei City 221, Taiwan.
Ying-Hui SuChildhood Cancer Foundation, Taipei 100, Taiwan.
Chih-Hsiang YuInstitute of Statistical Science Academia Sinica, Taipei 115, Taiwan.ORCID 0000-0001-5636-3415
Yung-Li YangDepartment of Pediatrics, National Taiwan University Hospital, Taipei 100, Taiwan.
Ssu-Chia WangTrineo Biotechnology Co., Ltd., New Taipei City 221, Taiwan.
Yi-Hsiu LinTrineo Biotechnology Co., Ltd., New Taipei City 221, Taiwan.
Yen-Ting ChenTrineo Biotechnology Co., Ltd., New Taipei City 221, Taiwan.ORCID 0000-0002-5586-7221
Jia-Yi LiTrineo Biotechnology Co., Ltd., New Taipei City 221, Taiwan.
Yen-Ting ChangDepartment of Clinical Laboratory Sciences and Medical Biotechnology, National Taiwan University, Taipei 100, Taiwan.
Su-Yu ChenTrineo Biotechnology Co., Ltd., New Taipei City 221, Taiwan.ORCID 0009-0009-1743-6891
Shu-Wha LinDepartment of Clinical Laboratory Sciences and Medical Biotechnology, National Taiwan University, Taipei 100, Taiwan.

Funding

Ministry of Science and Technology MOST 108-2321-B-002-069; MOST 109-2321-B-002-057
6 · The paper itself

Abstract

Gene therapy for hemophilia B offers the advantage of a single administration with sustained therapeutic effects. This study evaluated the systemic safety, efficacy, biodistribution, and immunogenicity of AAV8-FIX-TripleL, a recombinant adeno-associated virus type 8 (AAV8) vector encoding a modified factor IX (FIX) variant with increased activity. In this good laboratory practice (GLP)-compliant study, 180 male FIX-knockout hemophilia B mice were randomized into 12 groups (

Indexed as

DependovirusFactor IXGenetic TherapyGenetic VectorsHemophilia BAnimalsDisease Models, AnimalHumansMaleMiceMice, KnockoutTissue DistributionFactor IXAAV8FIXFIX-TripleLgene therapyhemophilia B

Identifiers

PMID40649847
PMCPMC12250290

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.