Evidence map›Paper›PMID 42137273›Full record

ArticleMolecular therapy. Advances2026

Manufacture of adeno-associated virus vectors by a novel human-derived cell line HAT and comprehensive evaluation of the vectors.

Yasuo Tsunaka, Mitsuko Fukuhara, Saki Shimojo, Aoba Matsushita, Takahiro Maruno, Sereirath Soth, Haruka Nishiumi, Mark Allen Vergara Rocafort, Toshie Kuwahara, Kenjiroo Matsumoto and 17 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Advances, 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. 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

27 authors.

Yasuo TsunakaDepartment of Biotechnology, Graduate School of Engineering, The University of Osaka, 2-1 Yamadaoka, Suita, Osaka 565-0871, Japan.
Mitsuko FukuharaDepartment of Biotechnology, Graduate School of Engineering, The University of Osaka, 2-1 Yamadaoka, Suita, Osaka 565-0871, Japan.
Saki ShimojoDepartment of Biotechnology, Graduate School of Engineering, The University of Osaka, 2-1 Yamadaoka, Suita, Osaka 565-0871, Japan.
Aoba MatsushitaU-Medico Inc., 2-1 Yamadaoka, Suita, Osaka 565-0871, Japan.
Takahiro MarunoU-Medico Inc., 2-1 Yamadaoka, Suita, Osaka 565-0871, Japan.
Sereirath SothDepartment of Biotechnology, Graduate School of Engineering, The University of Osaka, 2-1 Yamadaoka, Suita, Osaka 565-0871, Japan.
Haruka NishiumiDepartment of Biotechnology, Graduate School of Engineering, The University of Osaka, 2-1 Yamadaoka, Suita, Osaka 565-0871, Japan.
Mark Allen Vergara RocafortU-Medico Inc., 2-1 Yamadaoka, Suita, Osaka 565-0871, Japan.
Toshie KuwaharaU-Medico Inc., 2-1 Yamadaoka, Suita, Osaka 565-0871, Japan.
Kenjiroo MatsumotoU-Medico Inc., 2-1 Yamadaoka, Suita, Osaka 565-0871, Japan.
Kosei ShibataU-Medico Inc., 2-1 Yamadaoka, Suita, Osaka 565-0871, Japan.
Ryoji NakatsukaDepartment of Biotechnology, Graduate School of Engineering, The University of Osaka, 2-1 Yamadaoka, Suita, Osaka 565-0871, Japan.
Ryo AsahinaChitose Laboratory Corp., 3-2-1 Sakado, Takatsu-ku, Kawasaki, Kanagawa 213-0012, Japan.
Saho MizukadoMolecular and Cellular Glycoproteomics Research Group, Cellular and Molecular Biotechnology Research Institute, National Institute of Advanced Industrial Science and Technology (AIST), 1-1-1 Higashi, Tsukuba, Ibaraki 305-8565, Japan.
Yuuki FukaiDepartment of Neurophysiology & Neural Repair, Gunma University Graduate School of Medicine, Maebashi, Gunma 371-8511, Japan.
Tomoki TogashiDepartment of Biochemistry, Jichi Medical University School of Medicine, 3111-1 Yakushiji, Shimotsuke, Tochigi 329-0498, Japan.
Nemekhbayar BaatartsogtDepartment of Biochemistry, Jichi Medical University School of Medicine, 3111-1 Yakushiji, Shimotsuke, Tochigi 329-0498, Japan.
Kimitoshi TakedaU-Medico Inc., 2-1 Yamadaoka, Suita, Osaka 565-0871, Japan.
Atsushi KunoMolecular and Cellular Glycoproteomics Research Group, Cellular and Molecular Biotechnology Research Institute, National Institute of Advanced Industrial Science and Technology (AIST), 1-1-1 Higashi, Tsukuba, Ibaraki 305-8565, Japan.
Yuji KashiwakuraDepartment of Biochemistry, Jichi Medical University School of Medicine, 3111-1 Yakushiji, Shimotsuke, Tochigi 329-0498, Japan.
Yuki YamaguchiDepartment of Biotechnology, Graduate School of Engineering, The University of Osaka, 2-1 Yamadaoka, Suita, Osaka 565-0871, Japan.
Kazuaki NakamuraDepartment of Pharmacology, National Research Institute for Child Health and Development, 2-10-1 Okura, Setagaya-ku, Tokyo 157-8535, Japan.
Yugo HiraiChitose Laboratory Corp., 3-2-1 Sakado, Takatsu-ku, Kawasaki, Kanagawa 213-0012, Japan.
Hirokazu HiraiDepartment of Neurophysiology & Neural Repair, Gunma University Graduate School of Medicine, Maebashi, Gunma 371-8511, Japan.
Tsukasa OhmoriDepartment of Biochemistry, Jichi Medical University School of Medicine, 3111-1 Yakushiji, Shimotsuke, Tochigi 329-0498, Japan.
Takeshi OmasaDepartment of Biotechnology, Graduate School of Engineering, The University of Osaka, 2-1 Yamadaoka, Suita, Osaka 565-0871, Japan.
Susumu UchiyamaDepartment of Biotechnology, Graduate School of Engineering, The University of Osaka, 2-1 Yamadaoka, Suita, Osaka 565-0871, Japan.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Recombinant adeno-associated viruses (rAAVs) are prominent vectors in gene therapy. However, efficient, low-cost manufacture of the high-quality rAAVs that are necessary for clinical success remains challenging. Here, we report the manufacture of rAAVs using a novel human-derived suspended cell line, human amniotic epithelial cell line for gene and cell therapy (HAT). The transfection conditions for HAT were optimized for rAAV2, 5, and 9, with their titer and full particle ratio (EF ratio) as critical quality attributes. The EF ratios in HAT cell lysate for rAAV2, rAAV5, and rAAV9 were 46%, 17%, and 76%, respectively, which were all higher than the values from human embryonic kidney 293 (HEK293) cells. After purification of HAT-cell-produced rAAV9 by affinity and anion exchange chromatographies, the EF ratio reached 97%. Culture of rAAV9 in HAT cells in a 2-L bioreactor produced 7.7 × 10

Indexed as

AAVadeno-associated virusEF ratiogene therapyHAT cellmanufacturingpost-translational modificationquality attributeserotypetransduction

Identifiers

PMID42137273
PMCPMC13148930

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.