ArticleMolecular therapy. Methods & clinical development2022
Allometric-like scaling of AAV gene therapy for systemic protein delivery.
Article in Molecular therapy. Methods & clinical development, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 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
14 citing papers in PubMed, 19 citations in OpenAlex.
- Decoding Nonlinearities in AAV-Based Gene Therapy Using PBPK Modelling.The AAPS journal · 2026Article
- Study Designs and Crafting Endpoints for Gene Therapy Development Programs in Rare Disease: A Narrative Review.Advances in therapy · 2026Review
- From Radiocopper to Cold Copper: Mechanistic Modeling and Simulation to Define Clinical Response Criteria and Biomarkers for VTX-801 in Wilson Disease.CPT: pharmacometrics & systems pharmacology · 2026Article
- Development of VNX-101, an adeno-associated virus with less immunogenicity and efficient long-term expression of a CD19 T cell engager.Molecular therapy. Methods & clinical development · 2025Article
- Intratumoral Delivery of Genetically Engineered Anti-IL-6 Trans-signaling Therapeutics.Molecular biotechnology · 2025Article
- Designer Organs: Ethical Genetic Modifications in the Era of Machine Perfusion.Annual review of biomedical engineering · 2025Review
- Evaluation of Purification Methods for Minimizing Transgene Expression Background During Viral Manufacturing.Human gene therapy · 2025Article
- Chronopharmacology of AAV gene therapy in mice.microPublication biology · 2025Article
- Recombinant Adeno-Associated Virus Vectors for Gene Therapy of the Central Nervous System: Delivery Routes and Clinical Aspects.Biomedicines · 2024Review
- Subretinal AAV delivery of RNAi-therapeutics targetingMolecular therapy. Methods & clinical development · 2024Article
- Engineering viral vectors for acoustically targeted gene delivery.Nature communications · 2024Article
- Challenges, approaches and enablers: effectively triangulating towards dose selection in pediatric rare diseases.Journal of pharmacokinetics and pharmacodynamics · 2023Review
- Quantification of human mature frataxin protein expression in nonhuman primate hearts after gene therapy.Communications biology · 2023Article
- Toward lentiviral vectors for antiangiogenic ocular gene therapy.Molecular therapy. Methods & clinical development · 2023Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors at 1 institution in 1 country.
Funding
Abstract
The use of adeno-associated virus (AAV) as a gene delivery vehicle for secreted peptide therapeutics can enable a new approach to durably manage chronic protein insufficiencies in patients. Yet, dosing of AAVs have been largely empirical to date. In this report, we explore the dose-response relationship of AAVs encoding a secreted luciferase reporter to establish a mathematical model that can be used to predict steady-state protein concentrations in mice based on steady-state secretion rates
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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.