RNA repair for haemophilia A

Hengjun Chao, Christopher E. Walsh

Research output: Contribution to journalReview articlepeer-review

8 Scopus citations

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.

Original languageEnglish
Pages (from-to)1-8
Number of pages8
JournalExpert Reviews in Molecular Medicine
Volume8
Issue number1
DOIs
StatePublished - Jan 2006

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