ArticleGene therapy2004
A LacZ-based transgenic mouse for detection of somatic gene repair events in vivo.
Article in Gene therapy, 2004. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 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.
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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
9 citing papers in PubMed, 17 citations in OpenAlex.
- TIGER: A tdTomato in vivo genome-editing reporter mouse for investigating precision-editor delivery approaches.Proceedings of the National Academy of Sciences of the United States of America · 2025Article
- Review
- Cinnabarinic Acid-Induced Stanniocalcin 2 Confers Cytoprotection against Alcohol-Induced Liver Injury.The Journal of pharmacology and experimental therapeutics · 2022Article
- Novel reporter mouse models useful for evaluatingMolecular therapy. Nucleic acids · 2021Article
- Adeno-associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo.Hepatology (Baltimore, Md.) · 2010Article
- Gene targeting in vivo by adeno-associated virus vectors.Nature biotechnology · 2006Article
- A trial of somatic gene targeting in vivo with an adenovirus vector.Genetic vaccines and therapy · 2005Article
- Gene therapy progress and prospects: targeted gene repair.Gene therapy · 2005Review
- Implications of cell cycle progression on functional sequence correction by short single-stranded DNA oligonucleotides.Gene therapy · 2005Article
Corrections and comments
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Authors and funding
2 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Somatic gene repair of disease-causing chromosomal mutations is a novel approach for gene therapy. This method would ensure that the corrected gene is regulated by its endogenous promoter and expressed at physiological levels in the appropriate cell types. A reporter mouse, Gtrosa26(tm1Col), was generated by targeting a mutated LacZ gene to the Rosa26 locus in mouse embryonic stem (ES) cells. The LacZ gene contains a G to A point mutation, resulting in a Glu to Lys amino-acid substitution at position 461, which abrogates enzymatic activity. The gene is expressed in ES cells, primary embryonic fibroblasts, and in all tissues examined in the adult mouse, including the lung, liver, kidney, spleen, heart, brain and smooth muscle. This transgenic mouse will allow testing of gene repair strategies in vivo and identification of which cell types can be successfully targeted by chromosomal gene repair. Although low levels of gene repair were achieved in the ES cells used to generate the Gtrosa26(tm1Col) mouse, preliminary attempts at gene repair in vivo were unsuccessful, thus highlighting the difficulties that will have to be overcome to get this approach to work.
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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.