ArticleGene therapy2002
Application of SFHR to gene therapy of monogenic disorders.
Article in Gene therapy, 2002. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers, 2 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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
21 citing papers in PubMed, 2 syntheses or guidelines pooled it, 67 citations in OpenAlex.
- Gene therapy for sickle cell disease.The Cochrane database of systematic reviews · 2020Pooled it
- Gene therapy for sickle cell disease.The Cochrane database of systematic reviews · 2018Pooled it
- Use of genome-editing tools to treat sickle cell disease.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
- Multiple copies of a linear donor fragment released in situ from a vector improve the efficiency of zinc-finger nuclease-mediated genome editing.Gene therapy · 2014Article
- 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
- Small fragment homologous replacement: evaluation of factors influencing modification efficiency in an eukaryotic assay system.PloS one · 2012Article
- An update on targeted gene repair in mammalian cells: methods and mechanisms.Journal of biomedical science · 2011Review
- Nanoparticles deliver triplex-forming PNAs for site-specific genomic recombination in CD34+ human hematopoietic progenitors.Molecular therapy : the journal of the American Society of Gene Therapy · 2011Article
- 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
- Targeted gene insertion for molecular medicine.Journal of molecular medicine (Berlin, Germany) · 2008Review
- Cftr gene targeting in mouse embryonic stem cells mediated by Small Fragment Homologous Replacement (SFHR).Frontiers in bioscience : a journal and virtual library · 2008Review
- Site-specific base changes in the coding or promoter region of the human beta- and gamma-globin genes by single-stranded oligonucleotides.The Biochemical journal · 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
- Illegitimate DNA integration in mammalian cells.Gene therapy · 2003Review
- Sequence-specific modification of genomic DNA by small DNA fragments.The Journal of clinical investigation · 2003Review
- Targeted genetic repair: an emerging approach to genetic therapy.The Journal of clinical investigation · 2003Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Gene therapy treatment of disease will be greatly facilitated by the identification of genetic mutations through the Human Genome Project. The specific treatment will ultimately depend on the type of mutation as different genetic lesions will require different gene therapies. For example, large rearrangements and translocations may call for complementation with vectors containing the cDNA for the wild-type (wt) gene. On the other hand, smaller lesions, such as the reversion, addition or deletion of only a few base pairs, on single genes, or monogenic disorders, lend themselves to gene targeting. The potential for one gene targeting technique, small fragment homologous replacement (SFHR) to the gene therapy treatment of sickle cell disease (SCD) is presented. Successful conversion of the wt-beta-globin locus to a SCD genotype of human lymphocytes (K562) and progenitor/stem hematopoietic cells (CD34(+) and lin-CD38-) was achieved by electroporation or microinjection small DNA fragments (SDF).
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What OpenQuestion holds
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.