ArticleGene therapy2026
Metadata assessment of non-human primate studies of AAV9 uncovers potential tissue specific variation in expression efficiency.
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.
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
3 citing papers in PubMed.
- Noninvasive Focal Gene Delivery into the Cerebellum of Non-Human Primates using Focused Ultrasound.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- Gene therapy for liver diseases: methods, challenges and opportunities.Journal of nanobiotechnology · 2026Review
- Mechanisms of and mitigating strategies for cellular immune responses to CRISPR-associated nucleases in genome editing therapy.Frontiers in medicine · 2026Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Adeno-associated virus (AAV)-based gene therapies have garnered significant attention and investment due to their clinical success and potential to address underlying causes of many diseases. AAV vectors provide effective delivery of therapeutic genetic material to disease-relevant tissues. When evaluating safety and efficacy of recombinant AAV vectors, biodistribution profiles play a critical role in novel therapy development. Herein, a biodistribution metadata analysis was performed on ten studies involving 51 cynomolgus macaques (Macaca fascicularis). The macaques received a self-complementary or single-stranded AAV9 vector containing chicken ß-actin (CBA) or cytomegalovirus (CMV173) promoters expressing fluorescent reporters or a human SMN1 gene. These studies covered various routes of administration (ROA) including intravenous (IV), intracisternal magna (ICM), and lumbar puncture intrathecal (IT) injection. Metadata analysis of AAV9 biodistribution showed relatively uniform vector genome delivery throughout spinal cord tissues over multiple timepoints and ROAs. Moreover, decreased expression efficiency of viral DNA in liver was observed regardless of the ROA, macaque age, or viral construct used. To understand this trend, epigenetic profiling of tissue-localized AAV9 vector genome DNA was performed. Experimental evidence supports partial silencing and repression of transgene expression in macaque liver. These findings point to plausible strategies to consider in preclinical development of AAV9 mediated gene therapies.
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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.