ArticleMolecular therapy. Advances2026
Engineering novel AAV capsids by broadly attenuated and subsequent muscle-specific tropism in mice and NHPs.
Article in Molecular therapy. Advances, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
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.
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.
Who cites it
1 citing paper in PubMed.
- Mechanistic Insights into Poloxamer 188-Mediated Stabilization of AAV8 Capsids Using Coarse-Grained Molecular Dynamics.Pharmaceutical research · 2026Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
19 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Recombinant adeno-associated virus (rAAV) vectors are a potent gene delivery tool, but their clinical application is restricted by poor transduction of target tissues and off-target toxicity. To address this, we employed a two-step capsid engineering strategy: broad attenuation of rAAV tropism followed by peptide-driven tissue-specific retargeting. We first generated AAV.Zero1, a capsid with markedly reduced transduction across tissues, by VR swapping from AAV9 into AAV2. Introducing an R585A substitution (AAV.Zero2) partially restored the transduction, while deletion of residues 585-587 (AAV.Zero3) abolished it. By inserting a myogenic peptide into the AAV.Zero3 backbone, we produced a novel capsid (AAV.eM), which drove robust muscle-specific transgene expression with minimal off-target transduction in the liver, lung, brain, and kidney. This favorable profile was consistent across two mouse strains and non-human primates. AAV.eM mediated expression levels comparable to the leading myotropic vector, MyoAAV 4A, but exhibited a superior safety profile. Importantly, AAV.eM was able to functionally rescue a mouse model of Duchenne muscular dystrophy following systemic delivery of a micro-dystrophin gene. These results establish AAV.eM as an improved myotropic vector with enhanced specificity and proof of concept for a platform to create capsids with specific properties that translate across species by addition of peptides onto low transduction backbones.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
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.