Evidence map›Paper›PMID 42458007›Full record

ArticleNature medicine2026

Oligonucleotide-siRNA conjugate for SOD1 amyotrophic lateral sclerosis: a phase 1 trial.

Weiqi Chen, Lingling Jiang, Chunling Duan, Moorim Kang, Jinyi Ye, Ling Wang, Yuesong Pan, Hui Qu, Xiaoling Liao, Xuejiao Zhou and 7 more

Abstract readClinical Trial, Phase I
PubMed Full text at PMC 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

17 authors.

Weiqi Chen *Department of Neurology, Beijing Tiantan Hospital, Capital Medical University, Beijing, China.
Lingling Jiang *China National Clinical Research Center for Neurological Diseases, Beijing Tiantan Hospital, Capital Medical University, Beijing, China.ORCID http://orcid.org/0000-0003-4694-6419
Chunling Duan *Ractigen Therapeutics, Nantong, China.
Moorim Kang *Ractigen Therapeutics, Nantong, China.
Jinyi YeDepartment of Neurology, Beijing Tiantan Hospital, Capital Medical University, Beijing, China.
Ling WangDepartment of Neurology, Beijing Tiantan Hospital, Capital Medical University, Beijing, China.
Yuesong PanDepartment of Neurology, Beijing Tiantan Hospital, Capital Medical University, Beijing, China.ORCID http://orcid.org/0000-0003-3082-6789
Hui QuDepartment of Neurology, Beijing Tiantan Hospital, Capital Medical University, Beijing, China.
Xiaoling LiaoDepartment of Neurology, Beijing Tiantan Hospital, Capital Medical University, Beijing, China.
Xuejiao ZhouDepartment of Neurology, Beijing Tiantan Hospital, Capital Medical University, Beijing, China.
Yichen LiRactigen Therapeutics, Nantong, China.
Zubao GanRactigen Therapeutics, Nantong, China.
Junnan ChenRactigen Therapeutics, Nantong, China.
Ian SchachtRactigen Therapeutics, Nantong, China.
Robert F PlaceRactigen Therapeutics, Nantong, China.
Long-Cheng LiRactigen Therapeutics, Nantong, China. lilc@ractigen.com.ORCID http://orcid.org/0000-0002-3971-6335
Yilong WangDepartment of Neurology, Beijing Tiantan Hospital, Capital Medical University, Beijing, China. yilong528@aliyun.com.ORCID http://orcid.org/0000-0002-3267-0039

Funding

Beijing Municipal Science and Technology Commission No.Z231100004823036Beijing Nova Program 20230484245
6 · The paper itself

Abstract

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease partly caused by gain-of-function mutations in superoxide dismutase 1 (SOD1). Here we developed RAG-17, an siRNA-targeting SOD1, using an accessory oligonucleotide conjugate platform for enhanced central nervous system (CNS) delivery. Preclinically, RAG-17 rescued motor neuron degeneration, delayed disease progression, preserved motor function and extended survival in SOD1

Indexed as

Amyotrophic Lateral SclerosisOligonucleotidesRNA, Small InterferingSuperoxide Dismutase-1AdultAgedAnimalsFemaleHumansMacaca fascicularisMaleMiddle AgedMotor NeuronsOligonucleotidesRNA, Small InterferingSOD1 protein, humanSuperoxide Dismutase-1

Identifiers

PMID42458007
PMCPMC13375534

What Socratic holds

Textmetadata
LicenceCC BY-NC-ND
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 Socratic graph.