ArticleHuman gene therapy methods2016
Development of Optimized AAV Serotype Vectors for High-Efficiency Transduction at Further Reduced Doses.
Article in Human gene therapy methods, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers.
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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.
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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.
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Who cites it
18 citing papers in PubMed, 20 citations in OpenAlex.
- Deaths in gene therapy of Duchenne muscular dystrophy and other diseases: Underlying mechanisms and mitigating strategies.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Review
- Review
- Key Challenges and Optimization Practices for Non-clinical Evaluation of AAV based Gene Therapy for Central Nervous System Disease.Pharmaceutical research · 2025Review
- Shaping viral immunotherapy towards cancer-targeted immunological cell death.Frontiers in oncology · 2025Review
- Development of capsid- and genome-modified optimized AAVrh74 vectors for muscle gene therapy.Molecular therapy. Methods & clinical development · 2023Article
- Rationale and strategies for the development of safe and effective optimized AAV vectors for human gene therapy.Molecular therapy. Nucleic acids · 2023Review
- Delivery of CRISPR-Cas9 system for screening and editing RNA binding proteins in cancer.Advanced drug delivery reviews · 2022Review
- AAV3-miRNA vectors for growth suppression of human hepatocellular carcinoma cells in vitro and human liver tumors in a murine xenograft model in vivo.Gene therapy · 2021Article
- AAV vectors engineered to target insulin receptor greatly enhance intramuscular gene delivery.Molecular therapy. Methods & clinical development · 2020Article
- Development of a Clinical Candidate AAV3 Vector for Gene Therapy of Hemophilia B.Human gene therapy · 2020Article
- Impact of Natural or Synthetic Singletons in the Capsid of Human Bocavirus 1 on Particle Infectivity and Immunoreactivity.Journal of virology · 2020Article
- Engineering adeno-associated virus vectors for gene therapy.Nature reviews. Genetics · 2020Review
- Next Generation of Adeno-Associated Virus Vectors for Gene Therapy for Human Liver Diseases.Gastroenterology clinics of North America · 2019Review
- Extraction ofEvidence-based complementary and alternative medicine : eCAM · 2018Article
- Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models.Journal of translational medicine · 2017Article
- AAV Infection: Protection from Cancer.Human gene therapy · 2017Article
- Review
- Cellular transduction mechanisms of adeno-associated viral vectors.Current opinion in virology · 2016Review
Corrections and comments
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Authors and funding
4 authors at 2 institutions in 1 country.
Funding
Abstract
We have described the development of capsid-modified next-generation AAV vectors for both AAV2 and AAV3 serotypes, in which specific surface-exposed tyrosine (Y), serine (S), threonine (T), and lysine (K) residues on viral capsids were modified to achieve high-efficiency transduction at lower doses. We have also described the development of genome-modified AAV vectors, in which the transcriptionally inactive, single-stranded AAV genome was modified to achieve improved transgene expression. Here, we describe that combination of capsid modifications and genome modifications leads to the generation of optimized AAV serotype vectors, which transduce cells and tissues more efficiently, both in vitro and in vivo, at ∼20-30-fold reduced doses. These studies have significant implications in the potential use of the optimized AAV serotype vectors in human gene therapy.
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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.