Evidence map›Paper›PMID 41238908›Full record

ArticleGene therapy2026

AAV-mediated BDNF and GAS6 muscle delivery delays disease onset in SOD1

Yicong Le, Gongjie Liu, Shenzhe Wu, Marialaina Nissenbaum, Alexander W Kusnecov, Philip Furmanski, Raymond B Birge, Renping Zhou

Abstract read
In one paragraph

Article in Gene therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed, 1 pooled it
–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 synthesis or guideline pooled it.

  1. Pooled it
  2. Review
  3. Disruption of BDNF signalling in neuropathologies.Biochemical Society transactions · 2026
    Review
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

8 authors.

Yicong LeDepartment of Chemical Biology, Ernest Mario School of Pharmacy, Rutgers, The State University of New Jersey, Piscataway, NJ, USA.
Gongjie LiuPanathera Inc., Piscataway, NJ, USA.ORCID 0009-0003-5056-4218
Shenzhe WuDepartment of Chemical Biology, Ernest Mario School of Pharmacy, Rutgers, The State University of New Jersey, Piscataway, NJ, USA.ORCID 0009-0005-3988-8996
Marialaina NissenbaumDepartment of Psychology, School of Arts and Science, Rutgers, The State University of New Jersey, Piscataway, NJ, USA.
Alexander W KusnecovDepartment of Psychology, School of Arts and Science, Rutgers, The State University of New Jersey, Piscataway, NJ, USA.
Philip FurmanskiDepartment of Chemical Biology, Ernest Mario School of Pharmacy, Rutgers, The State University of New Jersey, Piscataway, NJ, USA.
Raymond B BirgeRutgers Cancer Institute of New Jersey, New Brunswick, NJ, USA.
Renping ZhouDepartment of Chemical Biology, Ernest Mario School of Pharmacy, Rutgers, The State University of New Jersey, Piscataway, NJ, USA. rzhou@pharmacy.rutgers.edu.ORCID 0000-0001-6985-6542

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Amyotrophic Lateral Sclerosis (ALS) is a fatal neurodegenerative disease, with limited treatments. Gene therapy offers an alternative strategy for treating a large portion of ALS patients, however, the disparate genetic alterations in ALS complicate the development of gene therapies. Tyrosine receptor kinase B (TRKB) and Tyro3 receptors are highly expressed in mouse spinal cord motor neurons, suggesting that their ligands, brain-derived neurotrophic factor (BDNF) and growth arrest-specific 6 (GAS6), respectively, are crucial for neuronal survival. In this study, we tested whether genetically induced and muscle tissue-specific expression of such survival-enhancing ligands would ameliorate symptom development in the SOD1

Indexed as

Amyotrophic Lateral SclerosisBrain-Derived Neurotrophic FactorDependovirusGenetic TherapyIntercellular Signaling Peptides and ProteinsAnimalsDisease Models, AnimalFemaleGene Therapy AgentsGenetic VectorsGrowth Arrest-Specific Protein 6HumansMaleMiceMice, TransgenicMotor NeuronsBrain-Derived Neurotrophic FactorGrowth Arrest-Specific Protein 6Intercellular Signaling Peptides and ProteinsSuperoxide Dismutase-1

Identifiers

PMID41238908
PMCPMC13472854

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.