ArticleFrontiers in medicine2025
Preclinical biodistribution and toxicology assessment of an AAV5-based subretinal modifier gene therapy for retinitis pigmentosa.
Article in Frontiers in medicine, 2025. 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.
- Safety of Adeno-Associated Viral Vectors in Gene Therapy: Mechanisms of Toxicity, Clinical Risks, and Strategies for Their Minimization.International journal of molecular sciences · 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
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Retinitis pigmentosa (RP) is a multifactorial disease caused by mutations in over 100 genes, affecting ~1 in 4,000 people worldwide, and is characterized by abnormalities in the rod and cone photoreceptors. Gene therapy approaches are promising and have been established to provide an unprecedented treatment option for genetic diseases caused by mutation(s) in a single gene. However, traditional gene therapy approaches are not pragmatic for RP due to its heterogeneous genetic background. To this end, modifier gene therapy is a unique approach in which a transgene can correct or rescue the detrimental effects caused by mutations in unrelated gene(s). Nuclear receptor subfamily 2 group E member 3 (NR2E3) is a nuclear hormone receptor that exhibits the characteristics of a modifier gene. In preclinical studies, subretinal delivery of NR2E3 rescued the RP phenotype by resetting the molecular pathways to restore normal ocular structure and function in multiple models of RP disease. For clinical studies, AAV5
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.