RNA repair for haemophilia A.
Expert Rev Mol Med
; 8(1): 1-8, 2006 Jan 10.
Article
em En
| MEDLINE
| ID: mdl-16401355
ABSTRACT
The mainstay of gene transfer studies is the use of wild-type cDNAs to effect phenotypic correction of diseases. However, this strategy is not feasible for genetic diseases caused either by mutations of large genes or by dominant-negative mutations, or where the regulation of the gene is critical. In this review, we will discuss a novel RNA reprogramming strategy - spliceosome-mediated RNA trans-splicing - where the pre-messenger RNA is modified by the splicing of two independent RNA species. The use of trans-splicing to effect phenotypic change in the hereditary bleeding disorder haemophilia A will be discussed.
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Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Precursores de RNA
/
Terapia Genética
/
Trans-Splicing
/
Hemofilia A
Limite:
Animals
/
Humans
Idioma:
En
Revista:
Expert Rev Mol Med
Assunto da revista:
BIOLOGIA MOLECULAR
Ano de publicação:
2006
Tipo de documento:
Article
País de afiliação:
Estados Unidos