ArticleHuman gene therapy2020
The Platform Vector Gene Therapies Project: Increasing the Efficiency of Adeno-Associated Virus Gene Therapy Clinical Trial Startup.
Article in Human gene therapy, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06630650 (A Single Center Prospective Natural History and Outcome Measure Validation Study of Congenital Myasthenic Syndromes), which is not on this map. Cited by 18 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.
A Single Center Prospective Natural History and Outcome Measure Validation Study of Congenital Myasthenic Syndromes
Who cites it
18 citing papers in PubMed.
- Bridging science and hope: the evolving story of gene therapy for neuromuscular diseases.Frontiers in cell and developmental biology · 2026Review
- Perspectives on the FDA platform technology designation program for the approval of gene therapies: a Swiss multi-stakeholder exploratory interview study.Orphanet journal of rare diseases · 2025Article
- Building Better Medicine: Translational Justice and the Quest for Equity in US Healthcare.The American journal of bioethics : AJOB · 2025Article
- Considerations for mRNA Product Development, Regulation and Deployment Across the Lifecycle.Vaccines · 2025Review
- Adeno-Associated Virus Gene Therapy Development: Early Planning and Regulatory Considerations to Advance the Platform Vector Gene Therapy Program.Human gene therapy · 2025Article
- When promising therapies are out of reach: Ethical responsibilities of stakeholders in gene therapy trials for rare disorders.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- En route towards a personalized medicine approach: Innovative therapeutic modalities for connective tissue disorders.Matrix biology : journal of the International Society for Matrix Biology · 2023Review
- Improving cell and gene therapy safety and performance using next-generation Nanoplasmid vectors.Molecular therapy. Nucleic acids · 2023Review
- Using Peptide Nucleic Acid Hybridization Probes for Qualitative and Quantitative Analysis of Nucleic Acid Therapeutics by Capillary Electrophoresis.Analytical chemistry · 2023Article
- Successfully Navigating Food and Drug Administration Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The National Institutes of Health Platform Vector Gene Therapy Experience.Human gene therapy · 2023Article
- Advancing translational science education.Clinical and translational science · 2022Review
- Global Regulatory and Public Health Initiatives to Advance Pediatric Drug Development for Rare Diseases.Therapeutic innovation & regulatory science · 2022Review
- Lipid-Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing Components.Molecular pharmaceutics · 2022Review
- Review
- Teaching principles of translational science to a broad scientific audience using a case study approach: A pilot course from the National Center for Advancing Translational Sciences.Journal of clinical and translational science · 2022Article
- The Role of Recombinant AAV in Precise Genome Editing.Frontiers in genome editing · 2021Review
- Integrating United States Biomanufacturing Across Vaccines and Therapeutics.NAM perspectives · 2021Article
- Ensuring Stakeholder Feedback in the Design and Conduct of Clinical Trials for Rare Diseases: ISCTM Position Paper of the Orphan Disease Working Group.Innovations in clinical neuroscienceReview
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
15 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
PubMed holds no abstract for this paper.
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.