ArticleGene therapy2001
Expression of DeltaF508 CFTR in normal mouse lung after site-specific modification of CFTR sequences by SFHR.
Article in Gene therapy, 2001. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 28 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
28 citing papers in PubMed, 77 citations in OpenAlex.
- Cas9/gRNA targeted excision of cystic fibrosis-causing deep-intronic splicing mutations restores normal splicing of CFTR mRNA.PloS one · 2017Article
- Impact of gene editing on the study of cystic fibrosis.Human genetics · 2016Review
- The Gene Targeting Approach of Small Fragment Homologous Replacement (SFHR) Alters the Expression Patterns of DNA Repair and Cell Cycle Control Genes.Molecular therapy. Nucleic acids · 2016Article
- TALENs Facilitate Single-step Seamless SDF Correction of F508del CFTR in Airway Epithelial Submucosal Gland Cell-derived CF-iPSCs.Molecular therapy. Nucleic acids · 2016Article
- Nanoparticles that deliver triplex-forming peptide nucleic acid molecules correct F508del CFTR in airway epithelium.Nature communications · 2015Article
- Nuclease-mediated double-strand break (DSB) enhancement of small fragment homologous recombination (SFHR) gene modification in human-induced pluripotent stem cells (hiPSCs).Methods in molecular biology (Clifton, N.J.) · 2014Article
- Systemic delivery of triplex-forming PNA and donor DNA by nanoparticles mediates site-specific genome editing of human hematopoietic cells in vivo.Gene therapy · 2013Article
- Sequence-specific correction of genomic hypoxanthine-guanine phosphoribosyl transferase mutations in lymphoblasts by small fragment homologous replacement.Oligonucleotides · 2010Article
- A comparison of synthetic oligodeoxynucleotides, DNA fragments and AAV-1 for targeted episomal and chromosomal gene repair.BMC biotechnology · 2009Article
- Cftr gene targeting in mouse embryonic stem cells mediated by Small Fragment Homologous Replacement (SFHR).Frontiers in bioscience : a journal and virtual library · 2008Review
- Limited restoration of cystic fibrosis lung epithelium in vivo with adult bone marrow-derived cells.American journal of respiratory and critical care medicine · 2006Article
- Reaction parameters of targeted gene repair in mammalian cells.Molecular biotechnology · 2005Article
- A LacZ-based transgenic mouse for detection of somatic gene repair events in vivo.Gene therapy · 2004Article
- Targeted correction of single-base-pair mutations with adeno-associated virus vectors under nonselective conditions.Journal of virology · 2004Article
- Branched oligonucleotides induce in vivo gene conversion of a mutated EGFP reporter.Gene therapy · 2003Article
- Sequence-specific modification of genomic DNA by small DNA fragments.The Journal of clinical investigation · 2003Review
- A comparison of gene repair strategies in cell culture using a lacZ reporter system.Gene therapy · 2003Article
- Targeted genetic repair: an emerging approach to genetic therapy.The Journal of clinical investigation · 2003Review
- Barriers to and new approaches for gene therapy and gene delivery in cystic fibrosis.Advanced drug delivery reviews · 2002Review
- Gene therapy progress and prospects: cystic fibrosis.Gene therapy · 2002Review
Corrections and comments
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Authors and funding
12 authors at 6 institutions in 2 countries.
Funding
Abstract
The development of gene targeting strategies for specific modification of genomic DNA in human somatic cells has provided a potential gene therapy for the treatment of inherited diseases. One approach, small fragment homologous replacement (SFHR), directly targets and modifies specific genomic sequences with small fragments of exogenous DNA (400-800 bp) that are homologous to genomic sequences except for the desired modification. This approach has been effective for the in vitro modification of exon 10 in the cystic fibrosis transmembrane conductance regulator (CFTR) gene in human airway epithelial cells. As another step in the development of SFHR for gene therapy, studies were carried out to target and modify specific genomic sequences in exon 10 of the mouse CFTR (mCFTR) in vivo. Small DNA fragments (783 bp), homologous to mCFTR except for a 3-bp deletion (DeltaF508) and a silent mutation which introduces a unique restriction site (KpnI), were instilled into the lungs of normal mice using four different DNA vehicles (AVE, LipofectAMINE, DDAB, SuperFect). Successful modification was determined by PCR amplification of DNA or mRNA-derived cDNA followed by KpnI digestion. The results of these studies showed that SFHR can be used as a gene therapy to introduce specific modifications into the cells of clinically affected organs and that the cells will express the new sequence.
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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.