ArticleGene therapy1999
Efficient CFTR expression from AAV vectors packaged with promoters--the second generation.
Article in Gene therapy, 1999. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 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.
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Who cites it
16 citing papers in PubMed, 31 citations in OpenAlex.
- Revolution of AAV in Drug Discovery: From Delivery System to Clinical Application.Journal of medical virology · 2025Review
- Viral Vectors, Animal Models, and Cellular Targets for Gene Therapy of Cystic Fibrosis Lung Disease.Human gene therapy · 2020Review
- Adeno-Associated Virus Serotype-Specific Inverted Terminal Repeat Sequence Role in Vector Transgene Expression.Human gene therapy · 2020Article
- Article
- Noninvasive optical activation of Flp recombinase for genetic manipulation in deep mouse brain regions.Nature communications · 2019Article
- Review
- The Development of a Viral Mediated CRISPR/Cas9 System with Doxycycline Dependent gRNA Expression for Inducible In vitro and In vivo Genome Editing.Frontiers in molecular neuroscience · 2016Article
- The production of viral vectors designed to express large and difficult to express transgenes within neurons.Molecular brain · 2015Article
- Optimization of AAV expression cassettes to improve packaging capacity and transgene expression in neurons.Molecular brain · 2014Article
- Generation of novel AAV variants by directed evolution for improved CFTR delivery to human ciliated airway epithelium.Molecular therapy : the journal of the American Society of Gene Therapy · 2009Article
- Packaging capacity of adeno-associated virus serotypes: impact of larger genomes on infectivity and postentry steps.Journal of virology · 2005Article
- Novel molecular approaches to cystic fibrosis gene therapy.The Biochemical journal · 2005Review
- A shortened adeno-associated virus expression cassette for CFTR gene transfer to cystic fibrosis airway epithelia.Proceedings of the National Academy of Sciences of the United States of America · 2005Article
- Current status of gene therapy for inherited lung diseases.Annual review of physiology · 2003Review
- Quantitative comparison of expression with adeno-associated virus (AAV-2) brain-specific gene cassettes.Gene therapy · 2001Article
- Repair of CFTR mRNA by spliceosome-mediated RNA trans-splicing.Gene therapy · 2000Article
Corrections and comments
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Authors and funding
6 authors at 2 institutions in 1 country.
Funding
Abstract
Gene therapy studies of cystic fibrosis (CF) have shown that AAV-based vector was efficient in transferring but not in expressing the CFTR cDNA in the target cells. The levels of CFTR gene expression were limited by the small packaging capacity of AAV because it had been difficult to package the CFTR cDNA with an efficient promoter. In the present study we have developed a new generation of AAV/CFTR vectors which contain efficient short promoters to express the CFTR gene in target cells. To do so, we reduced the size of the CFTR cDNA by determining the minimal untranslated regions required for expression of CFTR cDNA. We also identified short and efficient promoters that could be packaged with the down-sized CFTR cDNA into a novel AAV vector that had a maximal packaging capacity. Functional analyses showed that the new vectors were packaged efficiently and expressed higher levels of CFTR than a vector in which the CFTR gene was driven by the ITR sequence of AAV. Transduction of airway epithelial cells containing [symbol: see text] 508 mutation with the new vectors demonstrated efficient expression of the wild-type CFTR and correction of the CF phenotype. In contrast, no significant CFTR expression was detected in cells infected with the vector that express the CFTR gene from the ITR. These findings support the notion that the AAV can be developed into an efficient vector to transduce the CFTR gene and vectors expressing higher levels of CFTR from an efficient promoter should provide better efficacy for gene therapy of cystic fibrosis.
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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.