ArticleMolecular therapy : the journal of the American Society of Gene Therapy2025
AAV delivery of artificial miRNA targeting MAPT for the reduction of tau pathology in Alzheimer's disease.
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.
What it found
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Who cites it
3 citing papers in PubMed.
- Tau dysfunction in alzheimer's disease: molecular and cellular mechanisms, genetic modulation, and therapeutic perspectives.Molecular biology reports · 2026Review
- Dysregulation of the Tau-Microtubule-End-Binding Protein Axis in Alzheimer's Disease and Related Tauopathies.International journal of molecular sciences · 2026Review
- AAV-mediated gene therapy for Alzheimer's disease: neuroprotective mechanisms and translational challenges.Frontiers in aging neuroscience · 2026Review
Corrections and comments
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Authors and funding
14 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Tau reduction is a promising therapeutic approach with the potential to slow the progression of Alzheimer's disease. Here, we propose adeno-associated viral (AAV) delivery of an artificial miRNA (amiRNA) targeting the microtubule-associated protein tau (MAPT) mRNA for sustained tau reduction with a single therapeutic injection. Out of 22 initial designs, we identified potent, accurately processed, and highly specific amiRNA candidates. Our lead, amiR
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Registered trials
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