ArticleGene therapy2004
Targeted conversion of the transthyretin gene in vitro and 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 14 papers.
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Who cites it
14 citing papers in PubMed, 55 citations in OpenAlex.
- Hereditary Transthyretin-Related Amyloidosis: Genetic Heterogeneity and Early Personalized Gene Therapy.Biomedicines · 2022Review
- Amyloid Polyneuropathy and Myocardial Amyloidosis 10 Years after Domino Liver Transplantation from a Patient with a Transthyretin Ser50Arg Mutation.Internal medicine (Tokyo, Japan) · 2017Article
- Liver-targeted gene therapy: Approaches and challenges.Liver transplantation : official publication of the American Association for the Study of Liver Diseases and the International Liver Transplantation Society · 2015Review
- Gene therapy approach to FAP: in vivo influence of T119M in TTR deposition in a transgenic V30M mouse model.Gene therapy · 2014Article
- Recent advances in transthyretin amyloidosis therapy.Translational neurodegeneration · 2014Review
- Familial amyloidotic polyneuropathy: current and emerging treatment options for transthyretin-mediated amyloidosis.The application of clinical genetics · 2012Article
- Effects of atelocollagen formulation containing oligonucleotide on endothelial permeability.Journal of drug delivery · 2012Article
- Modified bases enable high-efficiency oligonucleotide-mediated allelic replacement via mismatch repair evasion.Nucleic acids research · 2011Article
- Enhanced transthyretin tetramer stability following expression of an amyloid disease transsuppressor variant in mammalian cells.The journal of gene medicine · 2009Article
- Single-stranded oligonucleotide-mediated in vivo gene repair in the rd1 retina.Molecular vision · 2007Article
- Leptomeningeal familial amyloidosis: A rare differential diagnosis of leptomeningeal enhancement in MRI.Journal of neurology · 2006Article
- Therapeutic potential of RNA interference against cancer.Cancer science · 2006Review
- 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
- Gene therapy progress and prospects: targeted gene repair.Gene therapy · 2005Review
Corrections and comments
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Authors and funding
15 authors at 5 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Familial amyloidotic polyneuropathy (FAP) is the common form of hereditary generalized amyloidosis and is characterized by the accumulation of amyloid fibrils in the peripheral nerves and other organs. Liver transplantation has been utilized as a therapy for FAP, because the variant transthyretin (TTR) is predominantly synthesized by the liver, but this therapy is associated with several problems. Thus, we need to develop a new treatment that prevents the production of the variant TTR in the liver. In this study, we used HepG2 cells to show in vitro conversion of the TTR gene by single-stranded oligonucleotides (SSOs), embedded in atelocollagen, designed to promote endogenous repair of genomic DNA. For the in vivo portion of the study, we used liver from transgenic mice whose intrinsic wild-type TTR gene was replaced by the murine TTR Val30Met gene. The level of gene conversion was determined by real-time RCR combined with mutant-allele-specific amplification. Our results indicated that the level of gene conversion was approximately 11 and 9% of the total TTR gene in HepG2 cells and liver from transgenic mice, respectively. Gene therapy via this method may therefore be a promising alternative to liver transplantation for treatment of FAP.
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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.