Modification of pre-mRNA splicing by antisense oligonucleotides

• Autorzy: Kole R.
• Języki publikacji: EN

Abstrakty

EN

Antisene oligonucleotides have been extensively studied as agents that inhibit the expression of undesirable genes in a sequence specific manner. Results reviewed in this article show that antisene oligonucleotides can also restore the expression of genes inactivated by mutations causing genetic diseases. In this novel application, antisene oligonucleotides block aberrant splice sites created by the mutations, forcing the spliceosomes to form at correct splice sites, thus restoring the proper splicing pathway and consequently the activity of the damaged gene.

Słowa kluczowe

EN

gene therapy; beta-globin; antisense oligonucleotide; man; gene expression; mRNA; genetic disease; oligonucleotide

• Wydawca: -
• Czasopismo: Acta Biochimica Polonica
• Rocznik: 1997
• Tom: 44
• Numer: 2
• Opis fizyczny: p.231-237,fig.

Twórcy

autor

Kole R.

• University of North Carolina, Chapel Hill, NC 27599, USA; E-mail: [e-mail]

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