Evidence map›Paper›PMID 42773379›Full record

ArticleScience China. Life sciences2026

Intraputaminal AAV9 co-delivery of AADC and GDNF in severe AADC deficiency: first-in-human pediatric dose-escalation study.

Feng Han, Pingping Gao, Youfeng Zhou, Tongtong Cui, Yichao Xu, Xi Fang, Min Shen, Suzhen Dong, Yumin Zhong, Yingyan Wang and 23 more

Abstract read
PubMed Publisher
In one paragraph

Article in Science China. Life sciences, 2026. 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

33 authors.

Feng Han *Department of Neurology, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, 200127, China.
Pingping Gao *Department of Neurosurgery, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, 200127, China.
Youfeng Zhou *Department of Neurology, Fujian Children's Hospital (Fujian Branch of Shanghai Children's Medical Center), College of Clinical Medicine for Obstetrics & Gynecology and Pediatrics, Fujian Medical University, Fuzhou, 350001, China.
Tongtong Cui *State Key Laboratory of Organ Regeneration and Reconstruction, Institute of Zoology, Chinese Academy of Sciences, Beijing, 100101, China.
Yichao XuDepartment of Rehabilitation, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, 200127, China.
Xi FangDepartment of Rehabilitation, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, 200127, China.
Min ShenDepartment of Rehabilitation, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, 200127, China.
Suzhen DongDepartment of Radiology, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, 200127, China.
Yumin ZhongDepartment of Radiology, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, 200127, China.
Yingyan WangDepartment of Neurology, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, 200127, China.
Lijun BuDepartment of Neurology, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, 200127, China.
Li ChenDepartment of Neurology, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, 200127, China.
Yuanyin XiaDepartment of Neurology, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, 200127, China.
Ruen YaoDepartment of Medical Genetics and Molecular Diagnostic Laboratory, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, 200127, China.
Nan BaoDepartment of Neurosurgery, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, 200127, China.
Bin XueDepartment of Anesthesiology, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, 200127, China.
Yingying TaoDepartment of Anesthesiology, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, 200127, China.
Jingjie GeDepartment of Nuclear Medicine/PET Center, Huashan Hospital, Fudan University, Shanghai, 200235, China.
Zizhao JuDepartment of Nuclear Medicine/PET Center, Huashan Hospital, Fudan University, Shanghai, 200235, China.
Yuhua ZhuDepartment of Nuclear Medicine/PET Center, Huashan Hospital, Fudan University, Shanghai, 200235, China.
Yihui GuanDepartment of Nuclear Medicine/PET Center, Huashan Hospital, Fudan University, Shanghai, 200235, China.
Chuantao ZuoDepartment of Nuclear Medicine/PET Center, Huashan Hospital, Fudan University, Shanghai, 200235, China.
Xinyan SunThe Center for Clinical Research Management, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, 200127, China.
Shan DingShanghai Vitalgen BioPharma Co., Ltd., Shanghai, 201210, China.
Gui CaoShanghai Vitalgen BioPharma Co., Ltd., Shanghai, 201210, China.
Xianfeng WangShanghai Vitalgen BioPharma Co., Ltd., Shanghai, 201210, China.
Li FengShanghai Vitalgen BioPharma Co., Ltd., Shanghai, 201210, China.
Hongxia MaoShanghai Vitalgen BioPharma Co., Ltd., Shanghai, 201210, China.
Hua LvShanghai Vitalgen BioPharma Co., Ltd., Shanghai, 201210, China.
Xiaoping ZhaoShanghai Vitalgen BioPharma Co., Ltd., Shanghai, 201210, China.
Wei LiState Key Laboratory of Organ Regeneration and Reconstruction, Institute of Zoology, Chinese Academy of Sciences, Beijing, 100101, China. liwei@ioz.ac.cn.
Jiwen WangDepartment of Neurology, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, 200127, China. wangjiwen@scmc.com.cn.
Yunhai SongDepartment of Neurosurgery, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, 200127, China. songyunhai3021@126.com.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare, severe inherited neurotransmitter disorder causing combined monoamine depletion and severe motor, autonomic, and developmental impairment. Building on established intraputaminal AAV-mediated AADC replacement, we evaluated a next-generation dual-mechanism strategy that couples restoration of dopamine synthesis with neurotrophic circuit support by delivering an AAV9 vector co-expressing human AADC and glial cell line-derived neurotrophic factor (GDNF) in children with genetically confirmed severe AADC deficiency. Four children (36-59 months; 1 female, 3 males) without head control at baseline received a single bilateral MRI-guided intraputaminal infusion at one of three dose levels (2.0×10

Indexed as

AADC deficiencyAAV9dopamineGDNFgene therapyrare disease

Identifiers

PMID42773379

What OpenQuestion holds

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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.