Evidence map›Paper›PMID 40518668›Full record

ArticleMolecular therapy : the journal of the American Society of Gene Therapy2025

Gene therapy with covalently closed-end AAV vector for spinal muscular atrophy.

Haolin Duan, Ciliu Zhang, Zhongliang Zhang, Xiaole Wang, Junping Zhang, Lifen Yang, Fang He, Leilei Mao, Li Yang, Zou Pan and 6 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing 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

3 citing papers in PubMed.

  1. Review
  2. Genetic medicines for epilepsy: unlocking new avenues for seizure control.Frontiers in bioengineering and biotechnology · 2026
    Review
  3. New hope for older SMA patients with next-generation self-complementary AAV gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    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

16 authors.

Haolin DuanDepartment of Pediatrics, Xiangya Hospital of Central South University, Chang Sha, Hunan Province 410008, China.
Ciliu ZhangDepartment of Pediatrics, Xiangya Hospital of Central South University, Chang Sha, Hunan Province 410008, China.
Zhongliang ZhangNikegen (Hangzhou) Company Limited, Hangzhou 310018, China.
Xiaole WangDepartment of Pediatrics, Xiangya Hospital of Central South University, Chang Sha, Hunan Province 410008, China.
Junping ZhangHerman B. Wells Center for Pediatric Research, Indiana University School of Medicine, Indianapolis, IN 46202, USA.
Lifen YangDepartment of Pediatrics, Xiangya Hospital of Central South University, Chang Sha, Hunan Province 410008, China.
Fang HeDepartment of Pediatrics, Xiangya Hospital of Central South University, Chang Sha, Hunan Province 410008, China.
Leilei MaoDepartment of Pediatrics, Xiangya Hospital of Central South University, Chang Sha, Hunan Province 410008, China.
Li YangDepartment of Pediatrics, Xiangya Hospital of Central South University, Chang Sha, Hunan Province 410008, China.
Zou PanDepartment of Pediatrics, Xiangya Hospital of Central South University, Chang Sha, Hunan Province 410008, China.
Renzhi HanHerman B. Wells Center for Pediatric Research, Indiana University School of Medicine, Indianapolis, IN 46202, USA.
Weiming WangNikegen (Hangzhou) Company Limited, Hangzhou 310018, China.
Dao PanDivision of Experimental Hematology & Cancer Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA.
Fei YinDepartment of Pediatrics, Xiangya Hospital of Central South University, Chang Sha, Hunan Province 410008, China.
Weidong XiaoHerman B. Wells Center for Pediatric Research, Indiana University School of Medicine, Indianapolis, IN 46202, USA. Electronic address: xiaow@iu.edu.
Jing PengDepartment of Pediatrics, Xiangya Hospital of Central South University, Chang Sha, Hunan Province 410008, China. Electronic address: pengjing@csu.edu.cn.

Funding

Advancing CNS drug delivery via epigenetic modulationR01NS132504 · NINDS · CINCINNATI CHILDRENS HOSP MED CTR · PI Dao Pan · 2023 to 2026
$2.5M
NINDS NIH HHS R01 NS132504
6 · The paper itself

Abstract

Covalently closed-end adeno-associated virus (cceAAV) vector is a new generation of self-complementary adeno-associated virus (scAAV) vector that does not utilize a mutant inverted terminal repeat (ITR) for vector production. Importantly, packaged genomes of these cceAAV vectors are markedly more intact than traditional scAAVs, which typically contain a large fraction of incomplete genomes, including many that lost their self-complementary configuration. Here, we report the first in-human experience with a cceAAV vector. High quality clinical grade cceAAV vector based on AAV serotype 9 (AAV9) was produced in 200 L of suspension 293 cells with a total yield of 4.3 × 10

Indexed as

DependovirusGenetic TherapyGenetic VectorsMuscular Atrophy, SpinalChild, PreschoolFemaleHEK293 CellsHumansInfantMaleSurvival of Motor Neuron 1 ProteinTreatment OutcomeSurvival of Motor Neuron 1 ProteinAAV vectorcceAAV vectorgene therapyscAAV vectorspinal muscular atrophy

Identifiers

PMID40518668
PMCPMC12848183

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.