ReviewPharmaceutical research2025
Key Challenges and Optimization Practices for Non-clinical Evaluation of AAV based Gene Therapy for Central Nervous System Disease.
Review in Pharmaceutical research, 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.
- Patient-derived tissue cultures complement neurospheres for preclinical evaluation of AAV-mediated gene delivery in glioblastoma.Journal of neuro-oncology · 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
4 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Diseases of the central nervous system represent a significant challenge in the field of medical research and clinical practice. The utilization of gene therapy technology represents a promising therapeutic strategy aimed at ameliorating diseases at the genetic level. Nonetheless, the presence of the blood-brain barrier (BBB) and challenges pertaining to delivery efficiency, expression site and level, along with the potential risks linked to overexpression and prolonged expression, raise considerable safety concerns. The design, production, delivery, and expression of adeno-associated virus (AAV) vectors significantly influence both clinical efficacy and safety considerations. This article examines the factors contributing to and potential strategies for addressing the low transduction efficiency associated with the blood-brain barrier, focusing on the optimization of vector design, delivery methods, specific expression, and distribution. The study examines the optimization of promoter synthesis, the application of machine learning algorithms for the enhancement of AAV capsid evolution, and the evaluation of various administration routes. Additionally, it explores innovative delivery methods, including mannitol intra-arterial delivery and focused ultrasound strategies, aimed at improving efficacy and safety. These initiatives offer valuable guidance and insights pertaining to gene therapies aimed at addressing neurodegenerative diseases and various disorders of the central nervous system.
Indexed as
Identifiers
41034677What 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.