Evidence map›Paper›PMID 42137272›Full record

ArticleMolecular therapy. Advances2026

Capsid-engineered AAV vector overcomes a key intracellular barrier and efficiently transduces spiral ganglion neurons in adult mice.

Jennifer Marx, Peixin Huang, Sereina O Sutter, Moritz Ertelt, Odett Kaiser, Jennifer Harre, Juliane Schott, Josephine Wilkes, Philipp Neek-John, Axel Rossi and 5 more

Abstract read
In one paragraph

Article in Molecular therapy. Advances, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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0cells of the map it votes in
0citing papers in PubMed
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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

15 authors.

Jennifer MarxInstitute of Experimental Hematology, Hannover Medical School, 30625 Hannover, Germany.
Peixin HuangDepartment of Otolaryngology Head and Neck Surgery, University of Kansas School of Medicine, Kansas City, KS 66160, USA.
Sereina O SutterInstitute of Virology, University of Zurich, Zurich, Switzerland.
Moritz ErteltInstitute for Drug Discovery, Leipzig University Faculty of Medicine, 04103 Leipzig, Germany.
Odett KaiserDepartment of Otolaryngology, Head and Neck Surgery, Hannover Medical School, 30625 Hannover, Germany.
Jennifer HarreDepartment of Otolaryngology, Head and Neck Surgery, Hannover Medical School, 30625 Hannover, Germany.
Juliane SchottInstitute of Experimental Hematology, Hannover Medical School, 30625 Hannover, Germany.
Josephine WilkesInstitute of Experimental Hematology, Hannover Medical School, 30625 Hannover, Germany.
Philipp Neek-JohnInstitute of Experimental Hematology, Hannover Medical School, 30625 Hannover, Germany.
Axel RossiInstitute of Experimental Hematology, Hannover Medical School, 30625 Hannover, Germany.
Athanasia WarneckeDepartment of Otolaryngology, Head and Neck Surgery, Hannover Medical School, 30625 Hannover, Germany.
Clara T SchoederInstitute for Drug Discovery, Leipzig University Faculty of Medicine, 04103 Leipzig, Germany.
Axel SchambachInstitute of Experimental Hematology, Hannover Medical School, 30625 Hannover, Germany.
Hinrich StaeckerDepartment of Otolaryngology Head and Neck Surgery, University of Kansas School of Medicine, Kansas City, KS 66160, USA.
Hildegard BüningInstitute of Experimental Hematology, Hannover Medical School, 30625 Hannover, Germany.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Hearing loss, the most prevalent sensory disorder, can result from pathology in various cochlear cell types. To develop effective gene therapy-based treatment strategies, vectors must be adapted to meet cell type-specific requirements. Here, we report a novel adeno-associated virus (AAV) capsid variant, AAV.MPI, which transduces spiral ganglion neurons (SGNs) in mature mouse cochleae with remarkable efficacy, even at low doses. This unique feature is mediated by a heptamer peptide, identified by a phage display peptide library screen, and inserted into the GH12/GH13 loop in each of the 60 subunits forming the mature AAV2 capsid. In addition to its change in vector tropism, AAV.MPI showed higher transgene expression efficacy. In line with this, uncoating assays revealed a more efficient release of vector genomes from AAV.MPI compared to AAV2 capsids, both

Indexed as

AAVAdeno-associated virusBDNFcapsid engineeringgene therapyinner earspiral ganglion neuronsuncoatingvector

Identifiers

PMID42137272
PMCPMC13148912

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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.