TY - JOUR
T1 - RNA repair for haemophilia A
AU - Chao, Hengjun
AU - Walsh, Christopher E.
PY - 2006/1
Y1 - 2006/1
N2 - 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.
AB - 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.
UR - http://www.scopus.com/inward/record.url?scp=33645829317&partnerID=8YFLogxK
U2 - 10.1017/S1462399406010337
DO - 10.1017/S1462399406010337
M3 - Review article
C2 - 16401355
AN - SCOPUS:33645829317
SN - 1462-3994
VL - 8
SP - 1
EP - 8
JO - Expert Reviews in Molecular Medicine
JF - Expert Reviews in Molecular Medicine
IS - 1
ER -