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Titolo:
Oligonucleotide-based gene correction strategies: Applications to neuromuscular and cardiovascular diseases
Autore:
Graham, IR; Beattie, SG; Hill, VJ; Dickson, G;
Indirizzi:
Univ London Royal Holloway & Bedford New Coll, Sch Biol Sci, Ctr Biomed Res, Egham TW20 0EX, Surrey, England Univ London Royal Holloway & Bedford NewColl Egham Surrey England TW20 0EX
Titolo Testata:
CROATIAN MEDICAL JOURNAL
fascicolo: 4, volume: 42, anno: 2001,
pagine: 467 - 472
SICI:
0353-9504(200108)42:4<467:OGCSAT>2.0.ZU;2-Z
Fonte:
ISI
Lingua:
ENG
Soggetto:
CHIMERIC RNA/DNA OLIGONUCLEOTIDES; RNA-DNA OLIGONUCLEOTIDE; SITE-DIRECTED MUTAGENESIS; DYSTROPHIN GENE; ANTISENSE OLIGONUCLEOTIDES; NUCLEOTIDE EXCHANGE; MAMMALIAN-CELLS; MESSENGER-RNA; MUTATION; REPAIR;
Keywords:
apolipoproteins; cardiovascular diseases; dystrophin; gene engineering; gene targeting; gene therapy; hyperlipidemia; muscular dystrophies; neuromuscular diseases; point mutation; risk factors; RNA, antisense; RNA splicing;
Tipo documento:
Article
Natura:
Periodico
Settore Disciplinare:
Clinical Medicine
Citazioni:
26
Recensione:
Indirizzi per estratti:
Indirizzo: Dickson, G Univ London Royal Holloway & Bedford New Coll, Sch Biol Sci, Ctr Biomed Res, Egham TW20 0EX, Surrey, England Univ London Royal Holloway & Bedford New Coll Egham Surrey England TW20 0EX
Citazione:
I.R. Graham et al., "Oligonucleotide-based gene correction strategies: Applications to neuromuscular and cardiovascular diseases", CROAT MED J, 42(4), 2001, pp. 467-472

Abstract

Gene augmentation is an attractive and viable approach in treatment of inherited diseases, despite its limitations, ie, the amount of viral genome inreplication-defective viral vectors is often too small for larger copy DNAs to be inserted. in addition, most viral vectors, when assessed in vivo, can elicit a host immune response, especially in cases of introduction of a gene copy where protein is completely absent, may have potential mutagenic effect on the host genome, or may be down-regulated. Therefore, alternativetherapeutic approaches are being investigated, such as chimeraplasty, in which a mutated allele that already exists in an affected individual can be corrected. Although the only gene defects that can be corrected by chimeraplasty are point mutations, and the correction frequncies are variable, it has been observed that intracellular delivery of oligonucleotides is likely to be more efficient than that of plasmid DNA or viral vectors, because corrected genes are expressed from their autologous promoters, ensuring thus correct spatial and temporal expression, and host immune response is not elicited. Another strategy in the therapy of inherited diseases, such as Duchenne muscular dystrophy, is the application of antisense RNA oligonucleotides, or splicomers, to the exclusion of exons whose mutation leads to production of deficient essential proteins. Here we report the recent progress made and problems encountered in each of these fields, and discuss the potential of nucleotide-based gene correction strategies in treatment of neuromuscular and cardiovascular diseases.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 20/09/20 alle ore 07:53:13