ReviewJournal of biomedical science2014
Improving clinical efficacy of adeno associated vectors by rational capsid bioengineering.
Review in Journal of biomedical science, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.
What it found
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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.
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
13 citing papers in PubMed, 24 citations in OpenAlex.
- Strategies for Evading Cellular Immunity Against Recombinant AAV Vectors in Gene Therapy.Current medical science · 2026Review
- Viral and non-viral cellular therapies for neurodegeneration.Frontiers in medicine · 2025Review
- Viral Vector-Based Gene Therapy for Epilepsy: What Does the Future Hold?Molecular diagnosis & therapy · 2024Article
- Deep Parallel Characterization of AAV Tropism and AAV-Mediated Transcriptional ChangesFrontiers in immunology · 2021Article
- Multiplexed Cre-dependent selection yields systemic AAVs for targeting distinct brain cell types.Nature methods · 2020Article
- Preparation and Administration of Adeno-associated Virus Vectors for Corneal Gene Delivery.Methods in molecular biology (Clifton, N.J.) · 2020Article
- Gene manipulation in liver ductal organoids by optimized recombinant adeno-associated virus vectors.The Journal of biological chemistry · 2019Article
- Seek and destroy: targeted adeno-associated viruses for gene delivery to hepatocellular carcinoma.Drug delivery · 2017Review
- Promise of adeno-associated virus as a gene therapy vector for cardiovascular diseases.Heart failure reviews · 2017Review
- Next-generation AAV vectors for clinical use: an ever-accelerating race.Virus genes · 2017Review
- Gene therapy as a potential tool for treating neuroblastoma-a focused review.Cancer gene therapy · 2016Review
- Hereditary Angioedema as a Metabolic Liver Disorder: Novel Therapeutic Options and Prospects for Cure.Frontiers in immunology · 2016Article
- E Pluribus Unum: 50 Years of Research, Millions of Viruses, and One Goal--Tailored Acceleration of AAV Evolution.Molecular therapy : the journal of the American Society of Gene Therapy · 2015Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
1 author at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Adeno associated vectors (AAV) have shown considerable promise to treat various genetic disorders in both preclinical and clinical settings mainly because of its safety profile. However, efficient use of AAV to deliver genes in immune-competent sites like muscles and liver requires very high doses which are associated with concomitant cellular immune response against the viral capsids leading to destruction of the transduced cells. Coupled with that, there are enough evidences that at high doses, AAV particles are subjected to increased cellular phosphorylation/uniquitination leading to proteasome mediated degradation and loss of the viral particles. The presence of preexisting immunity against AAV further adds on to the problem which is acting as a major roadblock to efficiently use it as a gene therapy vector in the clinics. To overcome this, rational bioengineering of AAV capsid becomes a prime tool by which specific amino acid residue(s) can be suitably modified/replaced by compatible residue(s) to create vectors having lower host immune response and higher intracellular trafficking rate. This article reviews the various aspects of rationally designing AAV capsids like by site-directed mutagenesis, directed evolution and combinatorial libraries which can create vectors having not only immune evasive property but also enhanced gene expression and transduction capability. One or more combinations of these strategies have strong potential to create novel vectors which will have suitable clinical efficiency even at a low dose.
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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.