Evidence map›Paper›PMID 42271071›Full record

ArticleNature medicine2026

Dual-target gene therapy in Parkinson's disease: a multicenter phase 1 trial.

Mengyue Niu, Jifeng Guo, Yuchao Yang, Qian Xu, Cheng Cheng, Hao Li, Ningdi Luo, Haiyan Zhou, Pingchen Zhang, Yuanyuan Li and 19 more

Abstract readMulticenter StudyClinical Trial, Phase I
PubMed Publisher
In one paragraph

Article in Nature medicine, 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

29 authors.

Mengyue Niu *Department of Neurology and Institute of Neurology, Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China.ORCID http://orcid.org/0000-0002-9714-593X
Jifeng Guo *Department of Neurology, Xiangya Hospital, Central South University, Changsha, China.
Yuchao Yang *Department of Neurology and Institute of Neurology, Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Qian XuDepartment of Neurology, Xiangya Hospital, Central South University, Changsha, China.
Cheng ChengBelief Biomed Inc., Shanghai, China.
Hao LiState Key Laboratory of Genetic Evolution & Animal Models, Kunming Institute of Zoology, Chinese Academy of Sciences, Kunming, China.ORCID http://orcid.org/0000-0002-3160-8719
Ningdi LuoDepartment of Neurology and Institute of Neurology, Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Haiyan ZhouDepartment of Neurology and Institute of Neurology, Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Pingchen ZhangDepartment of Neurology and Institute of Neurology, Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Yuanyuan LiDepartment of Neurology and Institute of Neurology, Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China.ORCID http://orcid.org/0000-0003-4967-3890
Ningyuan WangDepartment of Neurology and Institute of Neurology, Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Chunyi WangDepartment of Neurology and Institute of Neurology, Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Dianyou LiCenter for Functional Neurosurgery, Department of Neurosurgery, Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China.ORCID http://orcid.org/0000-0003-4212-4231
Zhengyu LinCenter for Functional Neurosurgery, Department of Neurosurgery, Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China.ORCID http://orcid.org/0000-0002-9797-9272
Peng HuangCenter for Functional Neurosurgery, Department of Neurosurgery, Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Zhiquan YangDepartment of Neurosurgery, Xiangya Hospital, Central South University, Changsha, China.
Zhuanyi YangDepartment of Neurosurgery, Xiangya Hospital, Central South University, Changsha, China.
Yanyan SongDepartment of Biostatistics, Clinical Research Institute, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Jing ZhengBelief Biomed Inc., Shanghai, China.
Wei JiangBelief Biomed Inc., Shanghai, China.
Zengmin DuBelief Biomed Inc., Shanghai, China.
Yuanxin QiBelief Biomed Inc., Shanghai, China.ORCID http://orcid.org/0009-0003-4865-699X
Huirong PengDepartment of Neurology, Xiangya Hospital, Central South University, Changsha, China.
Yuyan TanDepartment of Neurology and Institute of Neurology, Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Xintian HuState Key Laboratory of Genetic Evolution & Animal Models, Kunming Institute of Zoology, Chinese Academy of Sciences, Kunming, China.ORCID http://orcid.org/0000-0003-1216-4806
Xiao XiaoBelief Biomed Inc., Shanghai, China.
Lu ShenDepartment of Neurology, Xiangya Hospital, Central South University, Changsha, China. shenlu@csu.edu.cn.ORCID http://orcid.org/0000-0002-3393-8578
Jiaji LinResearch and Education Development Institute, Xi'an Jiaotong University, Xi'an, China. timklin@foxmail.com.ORCID http://orcid.org/0000-0002-3653-2389
Jun LiuDepartment of Neurology and Institute of Neurology, Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China. lj11128@rjh.com.cn.ORCID http://orcid.org/0000-0001-8300-8646

Funding

National Natural Science Foundation of China (National Science Foundation of China) 82230040National Natural Science Foundation of China (National Science Foundation of China) 82341251National Natural Science Foundation of China (National Science Foundation of China) 82401478
6 · The paper itself

Abstract

Restoring striatal dopamine synthesis is a promising gene therapy strategy for Parkinson's disease. Previous adeno-associated virus-mediated aromatic L-amino acid decarboxylase (AADC) monotherapies remain dependent on exogenous levodopa, whereas multigene delivery is constrained by strict adeno-associated virus packaging limits. A 'dual approach' targeting the two rate-limiting enzymes, tyrosine hydroxylase (TH) and AADC, offers the potential for autonomous dopamine synthesis. We report the 12-month primary safety and tolerability outcomes of a multicenter, open-label, dose-escalation, phase 1 trial evaluating BBM-P002, a new adeno-associated virus vector-AAVT42-codelivering constitutively active TH and AADC. Ten participants with moderate-to-advanced Parkinson's disease were enrolled and received bilateral intraputaminal infusions across doses of 4.0 × 10

Indexed as

Aromatic-L-Amino-Acid DecarboxylasesGenetic TherapyParkinson DiseaseTyrosine 3-MonooxygenaseAgedDependovirusDopamineFemaleGenetic VectorsHumansMaleMiddle AgedAromatic-L-Amino-Acid DecarboxylasesDopamineTyrosine 3-Monooxygenase

Identifiers

PMID42271071

What OpenQuestion holds

Textmetadata
Read underepoch 390

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.