Evidence map›Paper›PMID 41948127›Full record

ArticleMolecular therapy. Nucleic acids2026

Safety, efficacy, and distal nerve Schwann cell biodistribution in mice and NHPs to support translation of AAV9 RNAi therapy for CMT1A.

Marina Stavrou, Lindsay M Wallace, Merlin P Thangaraj, Noah K Taylor, Alexia Kagiava, Revekka Papacharalambous, Cynthia McAllister, Gloria Zender, Nizar Y Saad, M Bilal Bayazit and 8 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Nucleic acids, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. 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

18 authors.

Marina StavrouNeuroscience Department, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.
Lindsay M WallaceJerry R Mendell Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43210, USA.
Merlin P ThangarajJerry R Mendell Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43210, USA.
Noah K TaylorJerry R Mendell Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43210, USA.
Alexia KagiavaNeuroscience Department, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.
Revekka PapacharalambousNeuropathology Laboratory, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.
Cynthia McAllisterHistopathology Core at Nationwide Children's Hospital, Columbus, OH 43210, USA.
Gloria ZenderJerry R Mendell Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43210, USA.
Nizar Y SaadJerry R Mendell Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43210, USA.
M Bilal BayazitJerry R Mendell Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43210, USA.
Amanda HeslegraveDepartment of Neurodegenerative Disease, UCL Queen Square Institute of Neurology, London, UK.
Christina TryfonosVirology Department, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.
Jan RichterVirology Department, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.
Henrik ZetterbergDepartment of Neurodegenerative Disease, UCL Queen Square Institute of Neurology, London, UK.
Brian PriceArmatusBio Inc, Columbus, OH 43210, USA.
Rachel SalzmanArmatusBio Inc, Columbus, OH 43210, USA.
Kleopas A KleopaNeuroscience Department, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.
Scott Q HarperJerry R Mendell Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43210, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Charcot-Marie-Tooth (CMT) type 1A, the most common inherited demyelinating peripheral neuropathy, is caused by PMP22 gene duplication, leading to overproduction of PMP22 protein in Schwann cells. To treat CMT1A, we developed a PMP22 gene silencing therapy using adeno-associated viral vectors (AAV9) to deliver a therapeutic miRNA expression cassette (U6.miR871) via lumbar intrathecal administration. A single injection produced long-term miR871 expression, triggered selective RNA interference against the PMP22 mRNA, and subsequently lowered protein levels and improved disease manifestations in a humanized CMT1A model. To support clinical translation, we confirmed on-target specificity of miR871 for PMP22

Indexed as

AAVCharcot-Marie-Tooth type 1ACMT1Agene knockdowngene therapyintrathecal deliverymiRNAMT: Non-coding RNAsPMP22RNAiRNA interference

Identifiers

PMID41948127
PMCPMC13051718

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Registered trials

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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.