ArticleMolecular therapy : the journal of the American Society of Gene Therapy2014
Tropism-modified AAV vectors overcome barriers to successful cutaneous therapy.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 31 papers.
What it found
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Who cites it
31 citing papers in PubMed.
- AAVrh32.33 capsid demonstrates unexpected dermal tropism regardless of immunodominant epitope.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- Evaluation of the Efficacy of Transglutaminase 1 Gene Delivery by Adeno-Associated Virus into Rat and Pig Skin and Safety of ARCI Gene Therapy.International journal of molecular sciences · 2025Article
- Mapping administration route-dependent transduction profiles of commonly used AAV variants in mice by barcode amplicon sequencing.Molecular therapy. Methods & clinical development · 2025Article
- The evolving landscape of NF gene therapy: Hurdles and opportunities.Molecular therapy. Nucleic acids · 2025Review
- Development of CNS tropic AAV1-like variants with reduced liver-targeting following systemic administration in mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- AAV vectors: The Rubik's cube of human gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2022Review
- Identification of Broadly Applicable Adeno-Associated Virus Vectors by Systematic Comparison of Commonly Used Capsid VariantsHuman gene therapy · 2022Article
- Skin chronological aging drives age-related bone loss via secretion of cystatin-A.Nature aging · 2022Article
- High throughput screening of novel AAV capsids identifies variants for transduction of adult NSCs within the subventricular zone.Molecular therapy. Methods & clinical development · 2021Article
- Correction of recessive dystrophic epidermolysis bullosa by homology-directed repair-mediated genome editing.Molecular therapy : the journal of the American Society of Gene Therapy · 2021Article
- Pathogenetic Therapy of Epidermolysis Bullosa: Current State and Prospects.Bulletin of experimental biology and medicine · 2021Review
- Synthetic Biology: Emerging Concepts to Design and Advance Adeno-Associated Viral Vectors for Gene Therapy.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2021Review
- Novel AAV capsids for intravitreal gene therapy of photoreceptor disorders.EMBO molecular medicine · 2021Article
- Eliminating mesothelioma by AAV-vectored, PD1-based vaccination in the tumor microenvironment.Molecular therapy oncolytics · 2021Article
- Review
- Pre-arrayed Pan-AAV Peptide Display Libraries for Rapid Single-Round Screening.Molecular therapy : the journal of the American Society of Gene Therapy · 2020Article
- Selection of an Efficient AAV Vector for Robust CNS Transgene Expression.Molecular therapy. Methods & clinical development · 2019Article
- Therapeutic application of the CRISPR system: current issues and new prospects.Human genetics · 2019Review
- Adeno-associated virus vector as a platform for gene therapy delivery.Nature reviews. Drug discovery · 2019Review
- Capsid Modifications for Targeting and Improving the Efficacy of AAV Vectors.Molecular therapy. Methods & clinical development · 2019Review
Corrections and comments
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Authors and funding
15 authors.
Funding
Abstract
Autologous human keratinocytes (HK) forming sheet grafts are approved as skin substitutes. Genetic engineering of HK represents a promising technique to improve engraftment and survival of transplants. Although efficacious in keratinocyte-directed gene transfer, retro-/lentiviral vectors may raise safety concerns when applied in regenerative medicine. We therefore optimized adeno-associated viral (AAV) vectors of the serotype 2, characterized by an excellent safety profile, but lacking natural tropism for HK, through capsid engineering. Peptides, selected by AAV peptide display, engaged novel receptors that increased cell entry efficiency by up to 2,500-fold. The novel targeting vectors transduced HK with high efficiency and a remarkable specificity even in mixed cultures of HK and feeder cells. Moreover, differentiated keratinocytes in organotypic airlifted three-dimensional cultures were transduced following topical vector application. By exploiting comparative gene analysis we further succeeded in identifying αvβ8 integrin as a target receptor thus solving a major challenge of directed evolution approaches and describing a promising candidate receptor for cutaneous 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.